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Posts
Access to treatments
“A life-changing opportunity” – More boys accessing clinical trials through the DMD Hub
A community update about vamorolone and timeframes for patient access
A community update about vamorolone in North America
A community update about vamorolone in the United States
Accelerating potential treatments for DMD through repurposing
An update on Duchenne UK and PPMD’s $200,000 project to develop an accepted set of Patient Reported Outcomes
Clinical Trials Lectureship (Newcastle). End of project report summary
Data from wearable devices approved for use in Duchenne muscular dystrophy clinical trials
DMD Hub First Year Round-up
DMD Hub Gene Therapy meeting
DMD Hub Impact Report
DMD Hub Impact Report 2020
DMD Hub launches pilot project to improve access to research studies
DMD Hub Nurses Network
DMD Hub team expands
DMD Hub used as a showcase by the National Institute for Health Research
DMD INSPIRE 2019 Major Grant Call
DMD treatment Givinostat reaches important stage in European approval process
DMD treatment vamorolone approved in the US
Duchenne muscular dystrophy drug ataluren approved on the NHS
Duchenne UK and PPMD fund $200,000 to develop an accepted set of Patient Reported Outcomes
Duchenne UK and Project HERCULES announce new patient centred quality of life tool for Duchenne muscular dystrophy
Duchenne UK and the DMD Hub Gene Therapy Meeting: Summary Report
Duchenne UK announces £46,930 to fund the expansion of clinical trial capacity at Birmingham Heartlands Hospital
Duchenne UK announces a new grant of £163,637 to help improve the success rate of clinical trials
Duchenne UK attends the opening of new Clinical Research Facility at Leeds Children’s Hospital and announces £108,324 of funding for research posts
Duchenne UK awards £41,151 through the DMD Hub to Bristol Royal Hospital for Children, to help expand clinical trial capacity
Duchenne UK CEO speaks at the ABPI conference
Duchenne UK expands the DMD Hub to give DMD patients in London and the South-East more opportunities to access research
Duchenne UK funds DMD Hub Manager post for a further 4 years
Duchenne UK grants £130,173 to Royal Manchester Children’s Hospital to join the groundbreaking DMD Hub
Duchenne UK grants £63,618 to test whether TEMPOL can help reduce muscle cell damage and inflammation in DMD
Duchenne UK hosts an industry workshop on Economic Modelling for HTA
Duchenne UK introduces the DMD Hub and newly appointed Hub Manager: Emma Heslop.
Duchenne UK launches campaign to urge the MHRA to recognize FDA’s decision on Duvyzat
Duchenne UK launches new DMD Hub website: DMDHUB.ORG
Duchenne UK launches Project HERCULES to help patients get faster access to potentially life-changing treatments
Duchenne UK launches the DMD Clinical Research Hub
Duchenne UK statement on VPAS
Duchenne UK support development of Aparito digital measuring device
Duchenne UK Webinar: DMD Hub and Clinical Trial Update
Duchenne UK’s DMD HUB unveiled at World Muscle Society Meeting
Duchenne UK’s general election manifesto
Duchenne UK’s Project HERCULES Awarded Prestigious Rare Disease Award
Eight industry partners to support Phase 2 of Project HERCULES in 2019
Eli and the SMART Suit aim for Glastonbury!
EMA has recommended not to renew the marketing authorisation for Translarna in EU
EMA recommends approval of first ever treatment for all DMD patients
EMA’s negative decision on Translarna upheld
Emily Crossley’s ISPOR Blog
European Commission rejects European Medicines Agency’s recommendation not to renew Translarna
Fiona Lawrence is interviewed by Partnerships in Clinical Trials
First boy dosed on EMBARK gene therapy trial in the UK thanks to the DMD Hub Central Recruitment Project
First patients dosed in charity funded clinical trial, to test the safety and effectiveness of tamoxifen
First treatment for all patients with DMD approved in UK
First UK patient enrolled in DMD gene therapy trial at DMD Hub site
Gene therapy for DMD doesn’t meet primary endpoint in Phase 3 clinical trial
ICER Update
Improving data and decision-making in DMD preclinical research – latest update
Label for DMD gene therapy Elevidys expanded in the US
More clinical trials for DMD running in Glasgow thanks to the DMD Hub
New clinical trial posts funded at the DMD Hub
New DMD drug shows success in Phase 3 clinical trial
New neuromuscular consultant appointed to lead clinical trials as a result of DMD Hub funding
New recommendations for fair and timely access to DMD gene therapy clinical trials and treatments
New study to understand DMD patients’ and caregivers’ attitudes towards gene therapy launched
NICE release statement on continued access to ataluren (Translarna)
Nonprofit Foundations Launch Study to Improve Preclinical Research
Path to gene therapy approval in the UK – Sarepta and Roche’s SRP-9001
Project HERCULES in the media
Project HERCULES Webinar – Completion of Phase 1
Royal Approval on International Women’s Day
Sarepta and Roche’s SRP-9001 – FDA Advisory Committee meeting
Solid Biosciences Initiates Clinical Trial for Gene Transfer Candidate SGT-001 in Patients with Duchenne Muscular Dystrophy
Tamoxifen clinical trial, co-funded by Duchenne UK, is fully recruited, results expected early next year
Tamoxifen clinical trial, co-funded by Duchenne UK, now recruiting in the UK
Ten posts in place to support UK clinical trial capacity
Ten posts in place to support UK clinical trials
The DMD Hub Clinical Trial Coordinator Network
The Newcastle Workshop on Clinical Trials
Update on Sarepta and Roche’s SRP-9001 gene therapy
Vamorolone considered by NICE committee
Vamorolone given PIM designation for treatment in Duchenne muscular dystrophy
Vamorolone not recommended by NICE
Vamorolone, the first treatment for all patients with DMD, approved in European Union
Webinar about results of trial of gene therapy for DMD
Why we need a new approach to assessing rare diseases
Women and Duchenne – Fleur Chandler, Project HERCULES Chair
Article
A promising new horizon for DMD research
A rare day for a rare disease
Driving the research strategy and project pipeline with our new Scientific Advisory Board
How an international public/private partnership led to the first therapy for Duchenne muscular dystrophy
How can we better support patients and their families with the emotional and behavioural difficulties experienced in DMD?
Managing DMD over Christmas and New Year
Our new state-of-the-art data collection platform
Study shows potential for Lipid Nanoparticles to successfully deliver dystrophin gene to muscles
Why we need a new approach to assessing rare diseases
DMD research
‘Testosterone in DMD’ study published in the European Journal of Endocrinology
£1.5 million new fund launched to ‘Help the Heart’
17th International Conference on Duchenne and Becker Muscular Dystrophy
A big week for gene therapy
A community update about vamorolone and timeframes for patient access
A promising new horizon for DMD research
Accelerating potential treatments for DMD through repurposing
An update on our survey and workshop to discuss the use of re-purposed or off label medicines
An update on soy product research funded by Duchenne UK
Arrhythmias Update – October 2020
Breaking news: Duchenne UK backed drug shows success in clinical trial
Clinical Trials Lectureship (Newcastle). End of project report summary
Creating new muscle by using stem cells
Data from wearable devices approved for use in Duchenne muscular dystrophy clinical trials
Decentralised study to validate new home-based video assessment of DMD
Delay in European Access to Sarepta’s Exondys 51
Developing Gene Therapy For DMD
Disappointing findings in anti-inflammatory drug repurposing project
DMD Industry Roundup (Aug – Oct 2022)
DMD INSPIRE 2019 Major Grant Call
DMD Research roundup – Dec 2021 – Feb 2022
DMD Research Roundup (March – June 2022)
Dr Laurie Cave and Vasiliki Ioannidou appointed to our diet and nutrition project
Dr. Dada Pisconti presents preliminary neutrophil elastase inhibitor data at 2019 MDA conference
Driving the research strategy and project pipeline with our new Scientific Advisory Board
Duchenne UK and Parent Project Muscular Dystrophy Announce 2020 Joint Research Grant Call
Duchenne UK and Parent Project Muscular Dystrophy announce 2022 joint grant call
Duchenne UK and Parent Project Muscular Dystrophy award $350,000 (£260,000) to address immunological challenges of gene therapy in Duchenne Muscular Dystrophy
Duchenne UK and Parent Project Muscular Dystrophy award $500,000 to research into new muscle cell treatment for DMD
Duchenne UK and the DMD Hub Gene Therapy Meeting: Summary Report
Duchenne UK announces a grant of £53,000 to speed up the diagnosis of cardiac fibrosis in female carriers of Duchenne muscular dystrophy
Duchenne UK announces a new grant to Oxford University to investigate improved delivery of exon skipping drugs
Duchenne UK announces funding for a clinical trial to study the effects of Tamoxifen in Duchenne muscular dystrophy
Duchenne UK announces the first recipients of our Innovate Grant Call and grants £273,648 to the University of Western Australia to investigate Taurine, a nutraceutical, as a potential treatment for DMD
Duchenne UK attends the World Muscle Society 2018 Conference
Duchenne UK awards £45,000 to Leiden University Medical Center to investigate if Exon-skipping can treat the brains of patients with DMD
Duchenne UK commits $162,600 to support the development of a novel, minimally invasive biomarker to estimate lean muscle mass
Duchenne UK commits a further £60,000 to investigate soy products as a potential new treatment approach for DMD
Duchenne UK Community Webinar: Gene therapy and overcoming immunity challenges
Duchenne UK funds further research to understand the benefits of repurposed drugs in combination as a treatment for DMD
Duchenne UK funds hydrotherapy clinical trial for boys and young men with DMD
Duchenne UK funds new programme to help better understand the use of steroids and their impact on DMD
Duchenne UK funds new technique for finding effective DMD treatments
Duchenne UK funds Peptide Generation (PepGen) exon skipping project
Duchenne UK funds research into the repurposing of an anti-inflammatory drug to potentially promote muscle strength and movement
Duchenne UK funds research to further understand the benefits of dietary supplements as treatments for Duchenne Muscular Dystrophy.
Duchenne UK funds study to improve our knowledge of heart care in patients with DMD
Duchenne UK grants $500,000 to overcome immunity challenges in gene therapy
Duchenne UK grants £37,808 to research alternatives to night splints
Duchenne UK grants £63,618 to test whether TEMPOL can help reduce muscle cell damage and inflammation in DMD
Duchenne UK grants £655,000 to Evox Therapeutics to investigate new ways of delivering gene therapy to patients
Duchenne UK grants £82,405 to investigate the development of a potential new test system for drugs that impact fibrosis in DMD
Duchenne UK grants £86,460 to further understand the causes of heart disease in DMD
Duchenne UK Grants a further £780,000 to Tamoxifen Open Label Extension Study
Duchenne UK invests £1.25 million into new research of treatments for DMD
Duchenne UK invests £228,562 to address the use of testosterone as a treatment for Duchenne muscular dystrophy
Duchenne UK round up of 2017
Duchenne UK supports first study to examine Edasalonexent as a potential treatment for non-ambulant patients with DMD
Duchenne UK, MDA and PPMD collaborate on $686,500 grant to develop easier way to measure whether new Duchenne muscular dystrophy treatments are working
Elastase inhibitors as a potential treatment for DMD
EMA approves new measure for DMD drug trials that can replace the Six Minute Walking Test
Fibrosis Project Q&A
First boy dosed on EMBARK gene therapy trial in the UK thanks to the DMD Hub Central Recruitment Project
First patients dosed in charity funded clinical trial, to test the safety and effectiveness of tamoxifen
First Stage Results from the Metformin/L-Citruline Trial
First UK patient enrolled in DMD gene therapy trial at DMD Hub site
Focus On Caring For The Heart In Duchenne Muscular Dystrophy
Further investigation in to soy products
Gene editing start-up secures £3.4 million grant for DMD research
Gene therapy for DMD doesn’t meet primary endpoint in Phase 3 clinical trial
Haelan 951 research has promising results
Hannah Paish, PhD student on Duchenne UK project, wins 2nd place for her work
Helping muscle regeneration with repurposed medicines
How an international public/private partnership led to the first therapy for Duchenne muscular dystrophy
How can we better support patients and their families with the emotional and behavioural difficulties experienced in DMD?
Improving data and decision-making in DMD preclinical research – latest update
Investigating the benefits of Vitamin B3 (Nicotinamide Riboside)
Lay Summary: Cardiorespiratory Progression Over 5 Years and Role of Corticosteroids in Duchenne Muscular Dystrophy A Single-Site Retrospective Longitudinal Study
Montelukast: Repurposing of an anti-inflammatory drug to potentially promote muscle strength and movement
New clinical data for steroid-alternative drug Vamorolone announced
New DMD drug shows success in Phase 3 clinical trial
New Duchenne UK funding call for research into transformative treatments for DMD
New insights into muscle fibre loss in Duchenne muscular dystrophy
New Preclinical Data Support SGT-001 As A Novel Treatment Approach For Duchenne Muscular Dystrophy
New preclinical data supports SGT-001 as a novel treatment approach for Duchenne muscular dystrophy
New study to understand DMD patients’ and caregivers’ attitudes towards gene therapy launched
No strong evidence for tamoxifen as a treatment for Duchenne muscular dystrophy – TAM-DMD analysis concludes
Nonprofit Foundations Launch Study to Improve Preclinical Research
Our new project to improve diet and nutrition for people with DMD
Outcome measures for DMD clinical trials
Outcome measures for DMD clinical trials
Path to gene therapy approval in the UK – Sarepta and Roche’s SRP-9001
PepGen present latest data from new exon skipping therapy for Duchenne muscular dystrophy
Pfizer Doses First Patient Using Investigational Mini-Dystrophin Gene Therapy for the Treatment of Duchenne Muscular Dystrophy
Pfizer gene therapy for DMD doesn’t meet primary or secondary endpoints in Phase 3 clinical trial
Pfizer Terminates Domagrozumab (PF-06252616) Clinical Studies for the Treatment of Duchenne Muscular Dystrophy
Potential new therapy to treat inflammation and fibrosis
PPMD Conference Update: Blog 1: Gene therapy for DMD/BMD
Preclinical research into taurine as a potential DMD treatment identifies new biomarkers
Recommendations to bring gene therapy trials for Duchenne muscular dystrophy to the UK are published
Research Project Update: Biomarkers
Research results: Soy products show no benefit in reducing dystrophic symptoms in mdx mice
Results published from workshop with Psychosocial Project
Returning individual clinical trial data back to patients – VISION-DMD project update
ReveraGen Announces First Patient Enrollment in International Pivotal Trial of Vamorolone in Duchenne Muscular Dystrophy
ReveraGen BioPharma receives FDA Fast track designation for Vamorolone for the treatment of Duchenne muscular dystrophy
Santhera Files for Raxone
Sarepta and Roche’s SRP-9001 – FDA Advisory Committee meeting
Sarepta and Summit Enter Into Exclusive License Agreement
SAREPTA GENE THERAPY TRIAL PUT ON HOLD
Sarepta Therapeutics Announcement: Positive Preliminary Results from the First Three Children Dosed in the Phase 1/2a Gene Therapy Micro-dystrophin Trial
Sarepta Therapeutics announces hold to clinical study SRP-5051-201 (MOMENTUM) in the US
Serious adverse event on Pfizer’s Phase 1B gene therapy trial
SIDEROS Interim analysis FAQ
Simvastatin shows no impact on DMD progression
SMT C1100 Cleared for Phase 2 Trial
Solid Biosciences announce preliminary results and plans to dose escalate for IGNITE DMD
Solid Biosciences IGNITE DMD clinical trial begins
Solid Biosciences raises up to $50 million in Series C financing
Solid Biosciences report positive data from ongoing DMD gene therapy trial
Soy Products Study: Your Questions Answered
Statins as a potential treatment for DMD
Statins as a potential treatment for DMD
Study by RVC demonstrates potential of using gene therapy technology to treat DMD
Study finds some people with DMD may not need long-term testosterone supplement after pubertal induction
Study paves the way for improved detection of heart disease in female carriers of DMD
Study shows potential for Lipid Nanoparticles to successfully deliver dystrophin gene to muscles
Summary of paper published: Phase IIa trial in Duchenne muscular dystrophy shows vamorolone is a first-in-class dissociative steroidal anti-inflammatory drug
Summary Paper: Categorising trajectories and individual item changes of the North Star Ambulatory Assessment
Summit Announces PhaseOut DMD Did Not Meet Primary Endpoint
Summit Therapeutics announces that planned extension phase of their PhaseOut trial, will go ahead.
Summit Utrophin update
Tackling fibrosis and muscle regeneration
Tamoxifen clinical trial, co-funded by Duchenne UK, is fully recruited, results expected early next year
Tamoxifen clinical trial, co-funded by Duchenne UK, now recruiting in the UK
Taurine Project – an update on progress
Taurine Project Q&A
Technology used in COVID-19 vaccine to be used for Duchenne muscular dystrophy gene therapy research
Termination of funding of the Nutraceuticals Project with Reading University
The Solid Suit: Wearable support for patients with Duchenne muscular dystrophy (DMD)
Treat-NMD Conference Summary
Update on Duchenne UK’s grant to Evox Therapeutics
Update on Duchenne UK’s JAK Inhibitors project with AGADA Biosciences
Update on Sarepta and Roche’s SRP-9001 gene therapy
Update on Simvastatin as a potential treatment for DMD
Update on the clinical trial to investigate the effectiveness of Tamoxifen in Duchenne muscular dystrophy
Vamorolone given PIM designation for treatment in Duchenne muscular dystrophy
VISION-DMD Update
WAVE Life Sciences To Advance Nucleic Acid Therapies
We Invest £75,000 In Nutraceutical Research Project
Webinar about results of trial of gene therapy for DMD
Winner of #DreamWheelchair competition announced
Women and Duchenne – Dr Alessandra Gaeta, Director of Research
World Duchenne Day: Two New Gene Editing Projects
World Muscle Society 2021 – What we learnt
DMD tech
Decentralised study to validate new home-based video assessment of DMD
Duchenne UK collaborate with Whizz-Kidz to win £1 million from People’s Postcode Lottery Dream Fund
Duchenne UK joins forces with SRI International on the Smart Suit
Duchenne UK launch new competition to design dream wheelchair
Duchenne UK, SMA UK and University of Liverpool win £1.25m award for revolutionary mobility suit, funded by players of People’s Postcode Lottery
EMA approves new measure for DMD drug trials that can replace the Six Minute Walking Test
New app to prevent the wrong treatments being given to people with DMD in emergencies
Our new state-of-the-art data collection platform
The Solid Suit: Wearable support for patients with Duchenne muscular dystrophy (DMD)
Wheels of Change: BUILDING THE DREAM CHAIR
Duchenne UK news
100 up for Owen Farrell – AND JACK!
2021 impact report
Alex’s Wish Joins Duchenne UK as a “Partner Charity”
An update on our survey and workshop to discuss the use of re-purposed or off label medicines
An update on soy product research funded by Duchenne UK
Cheshire Dash raises more than £112,000 for Duchenne UK
Courier Post Logistics Ltd announces £1m support to Duchenne UK
DMD Hub First Year Round-up
DMD Hub Gene Therapy meeting
DMD Hub Impact Report 2020
DMD Hub team expands
DMD INSPIRE 2019 Major Grant Call
Dr. Dada Pisconti presents preliminary neutrophil elastase inhibitor data at 2019 MDA conference
Duchenne Awareness UK WDAD Interview with Duchenne UK’s co-founders
Duchenne Dash AT HOME raises £440,000!
Duchenne Now joins Duchenne UK
Duchenne UK announces £46,930 to fund the expansion of clinical trial capacity at Birmingham Heartlands Hospital
Duchenne UK announces the first recipients of our Innovate Grant Call and grants £273,648 to the University of Western Australia to investigate Taurine, a nutraceutical, as a potential treatment for DMD
Duchenne UK Attends the 12th UK Translational Research Conference for NMDs
Duchenne UK attends the opening of new Clinical Research Facility at Leeds Children’s Hospital and announces £108,324 of funding for research posts
Duchenne UK attends the World Muscle Society 2018 Conference
Duchenne UK awards £45,000 to Leiden University Medical Center to investigate if Exon-skipping can treat the brains of patients with DMD
Duchenne UK CEO joins the ABPI’s Patient Advisory Council
Duchenne UK CEO speaks at the ABPI conference
Duchenne UK commits $162,600 to support the development of a novel, minimally invasive biomarker to estimate lean muscle mass
Duchenne UK founders awarded OBEs in King’s Birthday Honours
Duchenne UK grants £10,000 to Decipha to support young people and their families living with Duchenne Muscular Dystrophy
Duchenne UK grants £655,000 to Evox Therapeutics to investigate new ways of delivering gene therapy to patients
Duchenne UK grants £82,405 to investigate the development of a potential new test system for drugs that impact fibrosis in DMD
Duchenne UK grants £86,460 to further understand the causes of heart disease in DMD
Duchenne UK hosts second Minecraft creative build competition for young people with DMD and their siblings
Duchenne UK is hiring!
Duchenne UK is looking for a Fundraising Manager
Duchenne UK is looking for a Project Manager
Duchenne UK launches
Duchenne UK launches campaign to urge the MHRA to recognize FDA’s decision on Duvyzat
Duchenne UK launches podcast: Decoding Duchenne
Duchenne UK launches START STOP October Challenge
Duchenne UK receives £10,000 donation from BGC and GFI Charity Day
Duchenne UK round up of 2017
Duchenne UK supporters invited to celebrate Captain Sir Tom’s achievements in special fundraising event
Duchenne UK welcomes new members to its Scientific Advisory Board
Duchenne UK, SMA UK and University of Liverpool win £1.25m award for revolutionary mobility suit, funded by players of People’s Postcode Lottery
Emily Crossley’s WMS blog
Festive MINI Electric embarks on charity tour, raising money for Duchenne UK
First patients dosed in charity funded clinical trial, to test the safety and effectiveness of tamoxifen
Focus On Caring For The Heart In Duchenne Muscular Dystrophy
Foodbuy extends charity partnership with Duchenne UK
Gene Therapy Meeting: Patient Perspectives
Heart specialist joins Duchenne UK scientific board
Helping muscle regeneration with repurposed medicines
Her Majesty The Queen renews patronage of Duchenne UK
HRH Duchess of Cornwall supports Duchenne UK
HRH The Duchess of Cornwall extends Presidency of Duchenne UK for a further five years
Impact Report 2020
Introducing our New Horizons conference
Jack Johnson on the ball at the Wheelchair Rugby League World Cup Final
Join the Duchenne UK team
Join the Duchenne UK team as Head of Communications
Join the Duchenne UK team as Project Coordinator
Kids, Jack And Rugby Stars Climb Snowdon
Marshall Wace names Duchenne UK as their charity of the year
Mother of Reinvention – Duchenne UK features in The Times
New £250,000 grant call for projects to support people with DMD as they enter adulthood and adult care
Opportunity to join Duchenne UK research team
Our boys are mascots for England Rugby at Six Nations
Our new state-of-the-art data collection platform
Outcome measures for DMD clinical trials
Owen Farrell and England Rugby help raise awareness for DMD at Six Nations
Owen Farrell and England Rugby help raise awareness for DMD at Six Nations
Rare Disease Day 2016
Rare Disease Day 2017
Santhera Pharmaceuticals, Summit Therapeutics, Catabasis Pharmaceuticals and Duchenne UK to host DMD Awareness Day
Saracens and Duchenne UK to raise funds for DMD Hub at Evelina London Children’s Hospital
Saracens Rugby Club and Duchenne UK join together to tackle Duchenne muscular dystrophy
Solid Biosciences Initiates Clinical Trial for Gene Transfer Candidate SGT-001 in Patients with Duchenne Muscular Dystrophy
Steve Mason’s 5 for 50 Marathon Challenge for Duchenne UK
Summary Paper: Categorising trajectories and individual item changes of the North Star Ambulatory Assessment
Team 3 Peaks for Project GO raise over £10,000 for Duchenne UK
The first Duchenne Dip raises £3,000
The Solid Suit: Wearable support for patients with Duchenne muscular dystrophy (DMD)
Update on the clinical trial to investigate the effectiveness of Tamoxifen in Duchenne muscular dystrophy
Vamorolone given PIM designation for treatment in Duchenne muscular dystrophy
We are recruiting for a full-time Fundraising Manager
Why Krishnan Guru-Murthy is presenting the BBC Radio 4 appeal for Duchenne UK – by Emily Reuben
Winner of #DreamWheelchair competition announced
World’s Strongest Boys
Events and fundraising
Berlin Marathon
Berlin Marathon
Cardiff Half Marathon
Cotswold Way Challenge
Dublin Marathon
Great North Run
Isle of Wight Challenge
Isle of Wight Challenge
Jurassic Coast Challenge
London Halloween Walk
London Summer Walk
London to Brighton Challenge
London to Brighton Challenge
London Winter Walk
Royal Parks Half Marathon
Santa in the City
Skydive for Duchenne UK
South Coast Challenge
Thames Bridges Trek
Thames Path Challenge
Three Peaks Challenge
Family and Friends Funds
Access to Life
Action 4 Arvin
Action for Zach
Archie’s Army
Archie’s March
Backing Jack
Bruce’s Battle Squad
Changing Charlie’s Future
Chasing Connor’s Cure
Cure4George
Defending William Against DMD
Doing it for Dexter
Edward Steam Team
Elliot’s Endeavours to End Duchenne
Ezra’s Mission to end Duchenne
Fight for Finn
Following Felix
For Felix
Help Harry
Helping Hayden
Henry’s Hurdles
Jack’s Aim
Jack’s Mission
Jacobi’s Wish
JJ’s Stronger Tomorrow
Joe’s Journey to End Duchenne
Josh’s Warriors
Kairo’s Krew
Life & Hope for Lenny
Lifting Louis
Love for Leon
Lucas Fighting Duchenne
Mac My Day
Matthew’s Mighty Mission
Mission Jensen
Moving Muscles for Marcus
Muscles for Mitchell
Oscar’s Duchenne Challengers
Project GO
Ralphy’s Fund
Rowan’s Raisers
Standing with Jack
Strength for Stanley
Team Callum
Team Dex
Team Felix
Team Thomas
The Lygo Family Fund
Together for Rhys
William’s Fund
News
£1.5 million new fund launched to ‘Help the Heart’
100 up for Owen Farrell – AND JACK!
2021 impact report
A community update about vamorolone in North America
A rare day for a rare disease
Alex’s Wish Joins Duchenne UK as a “Partner Charity”
An update on our survey and workshop to discuss the use of re-purposed or off label medicines
An update on soy product research funded by Duchenne UK
Cheshire Dash raises more than £112,000 for Duchenne UK
Courier Post Logistics Ltd announces £1m support to Duchenne UK
Decentralised study to validate new home-based video assessment of DMD
DMD Hub First Year Round-up
DMD Hub Gene Therapy meeting
DMD Hub Impact Report 2020
DMD Hub team expands
DMD INSPIRE 2019 Major Grant Call
DMD treatment Givinostat reaches important stage in European approval process
DMD treatment vamorolone approved in the US
Dr. Dada Pisconti presents preliminary neutrophil elastase inhibitor data at 2019 MDA conference
Duchenne Awareness UK WDAD Interview with Duchenne UK’s co-founders
Duchenne Dash AT HOME raises £440,000!
Duchenne Now joins Duchenne UK
Duchenne UK and Parent Project Muscular Dystrophy award $500,000 to research into new muscle cell treatment for DMD
Duchenne UK announces £46,930 to fund the expansion of clinical trial capacity at Birmingham Heartlands Hospital
Duchenne UK announces the first recipients of our Innovate Grant Call and grants £273,648 to the University of Western Australia to investigate Taurine, a nutraceutical, as a potential treatment for DMD
Duchenne UK Attends the 12th UK Translational Research Conference for NMDs
Duchenne UK attends the opening of new Clinical Research Facility at Leeds Children’s Hospital and announces £108,324 of funding for research posts
Duchenne UK attends the World Muscle Society 2018 Conference
Duchenne UK awards £45,000 to Leiden University Medical Center to investigate if Exon-skipping can treat the brains of patients with DMD
Duchenne UK CEO joins the ABPI’s Patient Advisory Council
Duchenne UK CEO speaks at the ABPI conference
Duchenne UK commits $162,600 to support the development of a novel, minimally invasive biomarker to estimate lean muscle mass
Duchenne UK founders awarded OBEs in King’s Birthday Honours
Duchenne UK grants £10,000 to Decipha to support young people and their families living with Duchenne Muscular Dystrophy
Duchenne UK grants £655,000 to Evox Therapeutics to investigate new ways of delivering gene therapy to patients
Duchenne UK grants £82,405 to investigate the development of a potential new test system for drugs that impact fibrosis in DMD
Duchenne UK grants £86,460 to further understand the causes of heart disease in DMD
Duchenne UK hosts second Minecraft creative build competition for young people with DMD and their siblings
Duchenne UK is hiring!
Duchenne UK is looking for a Fundraising Manager
Duchenne UK is looking for a Project Manager
Duchenne UK launches
Duchenne UK launches campaign to urge the MHRA to recognize FDA’s decision on Duvyzat
Duchenne UK launches podcast: Decoding Duchenne
Duchenne UK launches START STOP October Challenge
Duchenne UK receives £10,000 donation from BGC and GFI Charity Day
Duchenne UK round up of 2017
Duchenne UK supporters invited to celebrate Captain Sir Tom’s achievements in special fundraising event
Duchenne UK welcomes new members to its Scientific Advisory Board
Duchenne UK, SMA UK and University of Liverpool win £1.25m award for revolutionary mobility suit, funded by players of People’s Postcode Lottery
EMA approves new measure for DMD drug trials that can replace the Six Minute Walking Test
EMA has recommended not to renew the marketing authorisation for Translarna in EU
EMA recommends approval of first ever treatment for all DMD patients
Emily Crossley’s WMS blog
Festive MINI Electric embarks on charity tour, raising money for Duchenne UK
First patients dosed in charity funded clinical trial, to test the safety and effectiveness of tamoxifen
Focus On Caring For The Heart In Duchenne Muscular Dystrophy
Foodbuy extends charity partnership with Duchenne UK
Gene therapy for DMD doesn’t meet primary endpoint in Phase 3 clinical trial
Gene Therapy Meeting: Patient Perspectives
Heart specialist joins Duchenne UK scientific board
Helping muscle regeneration with repurposed medicines
Her Majesty The Queen renews patronage of Duchenne UK
HRH Duchess of Cornwall supports Duchenne UK
HRH The Duchess of Cornwall extends Presidency of Duchenne UK for a further five years
Impact Report 2020
Introducing our New Horizons conference
Jack Johnson on the ball at the Wheelchair Rugby League World Cup Final
Join the Duchenne UK team
Join the Duchenne UK team as Head of Communications
Join the Duchenne UK team as Project Coordinator
Kids, Jack And Rugby Stars Climb Snowdon
Marshall Wace names Duchenne UK as their charity of the year
Mother of Reinvention – Duchenne UK features in The Times
New £250,000 grant call for projects to support people with DMD as they enter adulthood and adult care
New DMD bone health leaflet for patients, parents and caregivers
New recommendations for fair and timely access to DMD gene therapy clinical trials and treatments
Opportunity to join Duchenne UK research team
Our boys are mascots for England Rugby at Six Nations
Our new project to improve diet and nutrition for people with DMD
Our new state-of-the-art data collection platform
Our programme to improve DMD psychosocial care
Outcome measures for DMD clinical trials
Owen Farrell and England Rugby help raise awareness for DMD at Six Nations
Owen Farrell and England Rugby help raise awareness for DMD at Six Nations
Preclinical research into taurine as a potential DMD treatment identifies new biomarkers
Rare Disease Day 2016
Rare Disease Day 2017
Santhera Pharmaceuticals, Summit Therapeutics, Catabasis Pharmaceuticals and Duchenne UK to host DMD Awareness Day
Saracens and Duchenne UK to raise funds for DMD Hub at Evelina London Children’s Hospital
Saracens Rugby Club and Duchenne UK join together to tackle Duchenne muscular dystrophy
Solid Biosciences Initiates Clinical Trial for Gene Transfer Candidate SGT-001 in Patients with Duchenne Muscular Dystrophy
Steve Mason’s 5 for 50 Marathon Challenge for Duchenne UK
Study finds some people with DMD may not need long-term testosterone supplement after pubertal induction
Summary Paper: Categorising trajectories and individual item changes of the North Star Ambulatory Assessment
Team 3 Peaks for Project GO raise over £10,000 for Duchenne UK
The first Duchenne Dip raises £3,000
The Solid Suit: Wearable support for patients with Duchenne muscular dystrophy (DMD)
Update on Sarepta and Roche’s SRP-9001 gene therapy
Update on the clinical trial to investigate the effectiveness of Tamoxifen in Duchenne muscular dystrophy
Vamorolone considered by NICE committee
Vamorolone given PIM designation for treatment in Duchenne muscular dystrophy
Vamorolone not recommended by NICE
We are recruiting for a full-time Fundraising Manager
Why Krishnan Guru-Murthy is presenting the BBC Radio 4 appeal for Duchenne UK – by Emily Reuben
Winner of #DreamWheelchair competition announced
World’s Strongest Boys
Our DMD care impact in 2023
Our DMD research impact in 2023
Breakthrough Vamorolone approval: Research we funded produces first ever drug for treatment of all people with DMD
Gene therapy trials: Secondary endpoints met in EMBARK trial and we are funding new research into optimising gene therapy for people with DMD
Our DMD Hub Central Recruitment Database: Making sure everyone with DMD has access to trials
Promising drug on its way: Global trials our DMD Hub ran in the UK shows Givinostat can slow DMD progression
Our DMD technology impact in 2023
Patient care & support
“Don’t wait for the fire”, warns DMD Care UK cardiologist
“A life-changing opportunity” – More boys accessing clinical trials through the DMD Hub
A rare day for a rare disease
Advocacy around the world! The Duchenne Caregivers Masterclass
Back to school COVID guidance for children with DMD, Oct 2020
Back to School update from Decipha
Back to School Webinar with Decipha
Calling all adults with Duchenne muscular dystrophy and parents of adults with DMD
Clinical guidance on risks from coronavirus for people with DMD
Coronavirus – Support for carers
COVID-19 spring booster: what we know so far
DMD Care UK Webinar – Adrenal Insufficiency, Bone & Puberty care recommendations
DMD Care UK Webinar – Steroid Stress Dosing Workshop
DMD Care UK: Adrenal Insufficiency, Bone & Puberty Information Leaflets & steroid dependent wristbands now available
DMD parents and caregivers urged to read new potentially life-saving recommendations
DMD Siblings Network – Meet Roan
Dr Laurie Cave and Vasiliki Ioannidou appointed to our diet and nutrition project
Duchenne muscular dystrophy in detail
Duchenne Research Fund partners with Duchenne UK to transform DMD psychosocial care
Duchenne UK and British Esports Association: online gaming tournament
Duchenne UK and Decipha continue to support young people with their education and careers
Duchenne UK and Joining Jack launch DMD Care UK – a major new initiative giving everyone living with Duchenne in the UK access to the best care
Duchenne UK and Pathfinders Neuromuscular Alliance to address lack of accessible housing
Duchenne UK authors on muscle biopsy paper published in Neuromuscular Disorders Journal
Duchenne UK CEO takes part in female carrier workshop
Duchenne UK chosen to join Aparito’s accelerator programme
Duchenne UK collaborate with Whizz-Kidz to win £1 million from People’s Postcode Lottery Dream Fund
Duchenne UK Community Webinar 2
Duchenne UK Community Webinar: Advice on steroids, adrenal suppression and stress dosing
Duchenne UK Community Webinar: Community Discussion: Lockdown and shielding
Duchenne UK Community Webinar: Education with Decipha
Duchenne UK Community Webinar: Facing Coronavirus Together: Tapping into the Resilience of the Rare Disease Community
Duchenne UK Community Webinar: Gene therapy and overcoming immunity challenges
Duchenne UK Community Webinar: Nutrition
Duchenne UK Community Webinar: Physiotherapy and exercise during lockdown
Duchenne UK continues to offer free educational support for children and young adults with DMD through Decipha
Duchenne UK funds hydrotherapy clinical trial for boys and young men with DMD
Duchenne UK grants £10,000 to Decipha to support young people and their families living with Duchenne Muscular Dystrophy
Duchenne UK grants £20,000 to develop an adult Neuromuscular Complex Care Centre in the North East
Duchenne UK hosts second Minecraft creative build competition for young people with DMD and their siblings
Duchenne UK launch In Case of Emergency App
Duchenne UK launch new competition to design dream wheelchair
Duchenne UK launch third Minecraft Creative Build Competition
Duchenne UK launches DMD Family Folder
Duchenne UK launches patient survey
Duchenne UK launches podcast: Decoding Duchenne
Duchenne UK publish new guide to growing up with DMD
Duchenne UK support development of Aparito digital measuring device
Duchenne UK update on Coronavirus (COVID-19)
Duchenne UK Webinar: Coronavirus (COVID-19) and Duchenne muscular dystrophy: Addressing the concerns of families in the UK
Duchenne UK, SMA UK and University of Liverpool win £1.25m award for revolutionary mobility suit, funded by players of People’s Postcode Lottery
Duchenne UK’s £80,000 grant to Decipha provides vital, free educational support for children and young adults with Duchenne Muscular Dystrophy
Duchenne UK’s offer to healthcare professionals – Emily Reuben
Emily Crossley writes for The Patient Group Handbook
Everyone aged 5 and over with DMD eligible for vaccination
Helping the DMD Community through virtual health appointments
How can we better support patients and their families with the emotional and behavioural difficulties experienced in DMD?
Janet Hoskin’s ‘A Guide to Duchenne Muscular Dystrophy’ published
JCVI: 16 to 17-year-olds should be offered vaccination
Join a psycho-social focus group
Meeting report: Improving access to the Standards of Care: roundtable discussion hosted by the Mayor of Greater Manchester – Andy Burnham, Joining Jack & Duchenne UK
National Strategy for Disabled People survey
New app to prevent the wrong treatments being given to people with DMD in emergencies
New COVID law: schools must provide remote learning
New DMD bone health leaflet for patients, parents and caregivers
Newcastle Information Day September 2018
NHS to GPs: vaccinate vulnerable 12 to 15-year-olds before school starts
NICE COVID-19 rapid guideline: critical care
Opportunity for adults with DMD to take part in research
Our new project to improve diet and nutrition for people with DMD
Our programme to improve DMD psychosocial care
Parent Information Day 2023
Patient and Caregiver perspectives on Duchenne Clinical Trials Survey Report
Patient Perspectives in DMD
Patient survey launched to better understand DMD care across the UK
PPMD Conference Update: Blog 2: Care Considerations
Project to improve psychosocial care for DMD now underway
Psychological and psychiatric support put in place for DMD patients and families
Raxone Patient Survey
RCPCH Hosts an Evening of Evidence for NMDs and Launches eLearning Tool to Improve Diagnosis
Results published from workshop with Psychosocial Project
Survey launched to better understand how prescription medicines and nutraceuticals are currently used by those living with DMD
The new rules and advice – guidance for the COVID alert system and going back to school
The Solid Suit: Wearable support for patients with Duchenne muscular dystrophy (DMD)
TREAT-NMD Statement on Stem Cell Tourism
Updated Care Consideration Guidelines and Family Guide for Duchenne
Updated Care Consideration Guidelines for Duchenne Published:
Updated clinical guidance on risks from coronavirus for people with DMD
Updated Family Guide for DMD
Updated school/college guidance for children with DMD, Nov 2020
Updates and advice from Duchenne UK’s cofounders on coronavirus (COVID-19)
Virtual reality trial to help children with DMD engage in physiotherapy at DMD Hub site
We have launched new guidance for better DMD respiratory care, which has been endorsed by the British Thoracic Society
Wheels of Change: BUILDING THE DREAM CHAIR
When clinical trials fail – Emily Crossley
Who are female (or manifesting) carriers?
World Duchenne Awareness Day 2020: Duchenne and the Brain
World Muscle Society issues new COVID-19 guidance for people with neuromuscular diseases
Policy and campaigning
A call to action – submit evidence for Exondys-51
An update from PPMD’s advocacy conference and gene therapy policy forum
BREXIT: the impact on medical innovation and patients with Duchenne Muscular Dystrophy in the UK
COVID-19 spring booster: what we know so far
DMD Pathfinders: pushing for DMD treatments for all ages
Duchenne muscular dystrophy drug ataluren approved on the NHS
Duchenne UK and DMD Pathfinders launch campaign for treatments for adults with DMD
Duchenne UK and Pathfinders Neuromuscular Alliance to address lack of accessible housing
Duchenne UK attend meeting for All-Party Parliamentary Group On Off-Patent Drugs
Duchenne UK CEO joins the ABPI’s Patient Advisory Council
Duchenne UK participates in the recent NICE/ NHS England consultation
Duchenne UK responds to the Life Sciences Industrial Strategy
Duchenne UK responds to UK Parliament consultation on Coronavirus (Covid-19) and the impact on people with protected characteristics
Duchenne UK statement on VPAS
Duchenne UK submits evidence to the parliamentary inquiry on the impact of COVID-19 on education and children’s services
Duchenne UK supports call for Life Sciences-Charity Partnership Fund
Duchenne UK’s general election manifesto
Everyone aged 5 and over with DMD eligible for vaccination
FDA delays decision on Eteplirsen
First Patients Enrolled in UK’s Early Access to Medicines Scheme for Santhera’s Raxone® in Duchenne Muscular Dystrophy (DMD)
Four charities call on Scottish Medicines Consortium to approve treatment for Duchenne muscular dystrophy
House of Lords Science and Technology Committee inquiry: The impact of Brexit on UK Duchenne muscular dystrophy research
JCVI: 16 to 17-year-olds should be offered vaccination
MHRA maintain a positive opinion on the Early Access to Medicines Scheme for Raxone
Minister’s open letter to SEND children and their parents/carers and families
National Disability Strategy ruled unlawful – appeal denied
NHS and PTC reach agreement on Translarna
NHS to GPs: vaccinate vulnerable 12 to 15-year-olds before school starts
NICE agrees managed access for ataluren (Translarna)
NICE COVID-19 rapid guideline: critical care
NICE release statement on continued access to ataluren (Translarna)
Our new report on the reality of living with Duchenne muscular dystrophy (DMD) in the UK
Path to gene therapy approval in the UK – Sarepta and Roche’s SRP-9001
Patient Group Consultative Forum at the MHRA covered the Early Access to Medicines Scheme and Brexit
Rare Disease Day 2016
Raxone Patient Survey
Santhera Pharmaceuticals, Summit Therapeutics, Catabasis Pharmaceuticals and Duchenne UK to host DMD Awareness Day
Santhera Provides Update on Timeline for Application of Raxone® in Duchenne Muscular Dystrophy in Europe
Sarepta and Roche’s SRP-9001 – FDA Advisory Committee meeting
The Impact Of Brexit On Rare Diseases
The MHRA invite patients and carers to contribute to discussions on whether to maintain positive scientific opinion for Raxone
Translarna – January 2021 update
Treat-NMD Conference Summary
Treatment for Duchenne muscular dystrophy accepted by Scottish Medicines Consortium
Update on Sarepta and Roche’s SRP-9001 gene therapy
Uncategorised
12 000 ft leap of faith!
18 years
A Dinner for the World’s Strongest Boys
Accelerated Access Review
Accelerating Research & Trial – test
Alan’s fundraising page for Connor
Alasdair Robertson
Alex Johnson: What trial is Jack taking part in?
Altering Muscle Environment To Change Stem Cell Behaviour
An evening with Jonny Wilkinson and Owen Farrell
An Update from Duchenne Parent Project Onulus Conference in Rome, Feb 2018
Andrew Nebel MBE – Chairman
Andy Farrell, OBE
Anti-Fibrotics
Assessing The Therapeutic Impact Of Cannabidiol
BGC Charity Day
BioMarin discontinues Kyndrisa (drispersen)
British London 10K
British London 10km Team Raises £10k
c
cake bake
Caroline Hazell
Cecilia Crossley
Chasing Connor’s Cure… Great South Run
Chasing Connors Cure resources
Chucking Myself Out Of A Perfectly Serviceable Plane for Felix
CJ Does 10k for Duchenne
Clinical Trial Capacity
Clinical Trial Lectureship
Collaborating With Other Funders
Combination Therapies
Congratulations to Joining Jack for winning the Dubai 7’s
Corporate Support
Correction Of DMD Mutations Using Genome Surgery
CRISPR
CRISPR: Gene editing advance
cup
current drugs intro
DCT funded Solid GT gains $42.5 investment
Decipha: patient and parent stories
Deepak Nambisan
Deutsche Bank Charity of the Year: Vote for Duchenne UK!
Discovering Biomarkers For Duchenne Muscular Dystrophy
Divyesh Popat
Donate
Donate a Day
Dr Anthony Hall
Dr David Bull
Dr Michael Gait
Dr. Eric Hoffman
Duchenne Dash 2015
Duchenne Forum
DUCHENNE INFORMATION DAY: PLEASE SAVE THE DATE
Duchenne Now
Duchenne UK and DMD Pathfinders on Muscle Owl
Duchenne UK announces a new grant to Oxford University to investigate improved delivery of exon skipping drugs
Duchenne UK Charity Film
Duchenne UK funds research into the repurposing of an anti-inflammatory drug to potentially promote muscle strength and movement
Duchenne UK funds study on promising COVID-19 anti-inflammatory drug to test its efficacy in Duchenne muscular dystrophy
Duchenne UK grants £10,000 to support young people and their families living with Duchenne Muscular Dystrophy
Duchenne UK hosts first Patient Information Day
Duchenne UK is investing £543,314 to fund 5 posts at the John Walton Muscular Dystrophy Research Centre and Royal Victoria Infirmary in Newcastle
Duchenne UK is looking for a Fundraising Manager
Duchenne UK is looking for a Project Manager
Duchenne UK is pleased to be supporting the upcoming TACT meeting
Duchenne UK Kids Triathlon
Duchenne UK launches
Duchenne UK launches the INNOVATE grant call, worth up to £1million
Duchenne UK receives Orphan Drug Designation for Metformin and L-Citrulline as a treatment for Duchenne Muscular Dystrophy
Duchenne UK responds to a new system to allow NHS patients faster access to ‘breakthrough’ medicines
Duchenne UK Triathlon
Duchenne UK welcomes new Director of Patient Engagement and Clinical Care
Duchenne UK’s INNOVATE grant call
Duchenneber
Duchenneber
Duchess of Cornwall Honey
Early Ambulatory
Early Ambulatory2
Eli Lilly and Company Joins cTAP
Eli Lilly and Company Joins cTAP
EMA renews approval of Ataluren (Translarna)
Emily speaks to the Medicines regulators
Emily writes for The Patient Group Handbook
EMPOWER New report
Empower: Data4Health
End Duchenne in 10
Engaging With Regulators
European Medicines Agency (EMA)
Family Fund A resources
Family Fund B resources
Family Fund C resources
Family Fund D resources
FDA AdComm for Eteplirsen
FDA delays decision on Eteplirsen
Fiona Lawrence
Fiona Lawrence is interviewed by Partnerships in Clinical Trials
Fiona Lawrence’s International Women’s Day blog: Swapping sectors is good…
Food & Drug Administration’s (FDA) Open Public Hearing
Foodbuy and Compass
football
For Eli
For Eli
frisbee
Funding Canadian based genome-editing technology CRISPR
Further Reading
Gene Editing (CRISPR)
Gene therapy breakthrough
Genetic Testing
Genome Editing To Repair DNA Duplications
Going electric for the 10th Duchenne Dash – LCH
Guðjón R. Óskarsson
Haelan 951 research has promising results
Hannah Becker
Harriet Moynihan
Head of Patient Data
Header floating buttons
Heart Specialist joins Duchenne UK Scientific Board
Hermione’s 10K
How Is Duchenne Inherited?
how is why?
How To Talk About End-Of-Life Care Planning
HRH Duchess of Cornwall supports Duchenne UK
HRH The Duchess of Cornwall – President
HT-100: A Drug To Treat Fibrosis And Inflammation
Imogen – 10K for Duchenne UK
Investigating inflammation in Duchenne muscular dystrophy
Jack’s Mission Gets Tough
january faq
January news
january page
John Bourke Heart Study, working with DMD
Join the Duchenne UK team
Join the Duchenne UK team as Head of Communications
Join the Duchenne UK team as Project Coordinator
Join the Duchenne UK team!
Jonathan Whitworth
Justine & Alex Latham
Katrina Ruthven
KiddieClimb
Kids, Jack And Rugby Stars Climb Snowdon
Kris Radlinski, MBE
Late Ambulatory
Late Ambulatory
Late Non-Ambulatory
Latest events
Learn About Clinical Trials
links
Lisa Kuhwald
Lisa Kuhwald joins Duchenne UK’s team as our Advocacy Support Officer
london
London Marathon
London Stone
Louise’s Fundraising page
Luca Buccella – We are the makers of our future
Maggie Wellington
Maria Hassard
Mary Nightingale
Mike Ramsden’s person of the year
Mike Wilkie, an inspirational fundraiser for Duchenne
Mumsnet Campaign: Join us to End Duchenne
Muscle Biopsy Survey
Newly Diagnosed
NHS and PTC reach agreement on Translarna
Nick Catlin
Nick Crossley
Non-Prescribed Supplements
nottm
Number of children on clinical trials doubles at Great Ormond Street Hospital, following funding through the DMD Hub
Our Impact
Our Impact (New)
Our Shop
Our Story
Outcome measures for DMD clinical trials
Partnerships in Clinical Trials Europe conference in Vienna
Patient centered outcome measurements in rare disease trials: Challenges and potential solutions
Patrons & Supporters
Pauline Clark
Peptide Generation (PepGen) project
Peter & Laura Williams
Peter Serafinowicz
Peter Williams
Pfizer Aquires Bamboo Therapeutics
Pfizer provides update on Myostatin trial
Positive results published showing the promise of using Human Artificial Chromosomes to deliver gene therapy
Pre-Register for the Duchenne Dash 2024
Prof. Annemieke Aartsma-Rus
Professor Dame Kay Davies
Professor Lord Smith of Clifton
Prudential Ride London-Surrey 100
PTC Therapeutics Provides Update on Translarna
Rare Disease Day 2017
Rebecca Burnett
Register your interest for the Duchenne Dash 2022
Repairing The Stem Cells Of People With DMD
Repurposing: Testing existing medicine for treatment of Duchenne Muscular Dystrophy
Results published from workshop with Psychosocial Project
ReveraGen BioPharma Receives FDA Fast Track Designation for Vamorolone for the Treatment of Duchenne Muscular Dystrophy
Robert McDonald M.D
Rugby Stars Unite For Duchenne
Running for Connor Bear
Santhera Pharmaceuticals has announced Raxone has been designated Promising Innovative Medicine (PIM)
Santhera’s Raxone® Receives First Positive EAMS Scientific Opinion from UK’s MHRA in Duchenne Muscular Dystrophy
Sara Parker Bowles
Sarah Alexander
Sarepta and Summit Enter Into Exclusive License Agreement
Sarepta Eteplirsen (Exondys 51) Managed Access Program Community Update
Sarepta European Community Update
Sarepta Therapeutics Announces EMA Validation of Eteplirsen Authorization Application
Sarepta Therapeutics Announces Positive Results in Its Study Evaluating Gene Expression, Dystrophin Production, and Dystrophin Localization in Patients with DMD
Sejal’s EURORDIS Winter School blog
Skydive
SMT C1100 Cleared for Phase 2 Trial
Solid Biosciences raises up to $50 million in Series C financing
Stages Intro
Stem Cells
steve
Study shows potential for Lipid Nanoparticles to successfully deliver dystrophin gene to muscles
Submit your personal stories about Ataluren/Translarna and help our friends in the USA
Summit Therapeutics announces that planned extension phase of their PhaseOut trial, will go ahead.
Summit Therapeutics Find New Biomarkers for DMD
Summit Therapeutics plc : Interim 24-Week Data from PhaseOut DMD
Summit update on Phase 2 Trial for Ezumitrod
Summit Utrophin Update
TACT
Team Felix Kiddie Climb 2016
Team Felix raise £57,000 at FeFest
Team Felix resources
TEAM SLK
Ten Posts in place to support UK Clinical Trial Capacity
Ten Posts In Place To Support UK Clinical Trials
test
test
test snippet
The Biological Process Of Muscle Fibre Death
The British 10K
The Campaign For Early Access To Medicines
The Duchenne Dash 2016
The Duchenne Dash 2019 by Ben Monro-Davies
The first Duchenne Dip raises £3,000
The Newcastle Plan
The Newcastle Workshop on Clinical Trials
this is a test
this is a test page
Tim Peake Tweets Us From Space
TJ runs the Vitality London 10k for Duchenne UK
TotallyMoney name us charity of the year
Translarna meeting news
Treat NMD
TREAT-NMD & TACT
Treatment For The Heart
TRI 4 DUCHENNE
Tri4Duchenne
Tri4Duchenne
Triathlon
Understanding Clinical Trials
Utrophin modulation – SMT C1100
Utrophin Modulators
Vici Richardson
Virtual Wigan Bike Ride 2020
VISION-DMD: Interview with Alex Johnson
water bottle
WCRS supporting World’s Strongest Boys
We Fund New Therapeutic Approach
What are the causes of DMD?
Working With Industry
xmas
xmas collection
xmas resources
xmas tree 43