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DMD research

‘Testosterone in DMD’ study published in the European Journal of Endocrinology
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
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. Dada Pisconti presents preliminary neutrophil elastase inhibitor data at 2019 MDA conference
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 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
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
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 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
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 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
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 paves the way for improved detection of heart disease in female carriers of DMD
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
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

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