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Duchenne UK launches


Ambitious new research charity, Duchenne UK, launches with a mission to end muscle wasting disease

Two of the largest UK charity funders of research to end Duchenne muscular dystrophy have come together to form Duchenne UK, a charity dedicated to funding and accelerating research and treatments solely for Duchenne muscular dystrophy (DMD).

Joining Jack and the Duchenne Children’s Trust have spent more than £4 million in the last three years on direct grants for research and clinical trials, and have now come together to form Duchenne UK, to drive momentum to delivering treatments to help this generation of those with DMD.

Duchenne UK's aim is to find treatments and a cure for this disease, by funding and accelerating research and clinical trials, to help hundreds of thousands of patients around the world.

Their collaborative track record is impressive: they have co-funded two successful Phase 1 clinical trials; they were early stage investors in Solid GT, a US based biotech company advancing gene therapies for DMD; and have funded 13 other research projects. In addition, they worked with other UK patient organisations to raise £1.2 million to fund 13 clinical research posts at leading muscle clinics.

Duchenne UK is run by Emily Crossley, Founder and Director of Duchenne Children’s Trust, and Alex Johnson, Founder and Director of Joining Jack.

Emily says, "We are delighted to cement an already strong and dynamic relationship into one organisation, Duchenne UK. We believe in the power of science and medicine to change lives. And of the strength of communities like ours to move mountains. Together we can change the future for everyone living with Duchenne muscular dystrophy".

Joining Jack will remain as a powerful and uniting fundraising brand for Duchenne UK.

Alex Johnson says, "Duchenne UK will have one Scientific Advisory Board, one Patient Advisory Board, and one coherent and ambitious vision over, one, two and five years to accelerate and fund research. We will continue to engage with companies and regulators, to ensure that patients with Duchenne are given access to safe, effective treatments as soon as possible."

Duchenne UK will continue to work closely with other charities to advance research, including the Duchenne Research Fund.

Kerry Rosenfeld, one of the founders of the Duchenne Research Fund says; "These are exciting times for research in Duchenne. DRF is so proud to work closely with, and complement Duchenne UK, to help achieve our shared joint mission of curing Duchenne for all patients, forever."

Alex and Emily are highly focused and driven patient advocates. They have both given evidence at public hearings at the Food and Drug Administration (FDA) in Washington DC. They sit on the Medicines and Healthcare Products Regulatory Agency's (MHRA) Patient Group Consultative Forum. Alex Johnson is a Eurordis trained patient advocate, and a board member of United Parent Project Muscular Dystrophy.

Fiona Lawrence has recently joined Duchenne UK as Director of Research and Clinical Development to help drive the charity's ambitious plans for 2016.

Professor Volker Straub, the Harold MacMillan Professor of Medicine at Newcastle University says; "In rare and devastating diseases like Duchenne muscular dystrophy, working together isn’t an option but a prerequisite, to accelerate the elaboration of more effective treatments that patients and families are eagerly waiting for. The clinical and research community welcomes the collaboration and partnership with Duchenne UK, to address the needs of patients with Duchenne muscular dystrophy together."

Duchenne muscular dystrophy is a desperately cruel muscle wasting disease that mainly affects boys. It is the most common fatal genetic disease to affect children. Treatment options are limited, and life expectancy is late 20s.

The build of the website was funded with a grant from the Transform Foundation.

Published on 6 May 2016

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