Welcome to Duchenne UK
JOINING Jack and the Duchenne Children's Trust are two of the UK’s biggest charity funders of research to end Duchenne Muscular Dystrophy, and we have come together to form Duchenne UK, the largest charity in the UK dedicated to funding and accelerating research and treatments solely for Duchenne Muscular Dystrophy (DMD).
Our aim is to find treatments and a cure for this disease, by funding and accelerating research and clinical trials, ending the suffering of thousands of patients around the world.
Joining Jack and the Duchenne Children’s Trust have between them, spent more than £4 million in the last three years on direct grants for research and clinical trials, and are now coming together to form Duchenne UK, to drive momentum to delivering treatments to help this generation of those with DMD.
They are building on an impressive collaborative record. They co-funded two successful Phase 1 clinical trials, and provided seed funding for a US based biotech’s gene therapy programme, run by Solid GT, which is on track to enter the clinical trial phase in the next 12 months. They are also funding 13 other research projects. In addition they worked with other UK patient organisations in the UK to raise £1.2 million to fund 13 clinical research posts at leading muscle centres in the UK.
Duchenne UK has a pioneering and ambitious research approach. On certain projects, we create a 'multiplier effect'. Duchenne UK provides research spend to prove a drug is ready for trial, which is then a catalyst for additional investment. They have had two successful projects where $6million of seed funding from the charity attracted $54m in further funding from investors and government funding bodies.
Duchenne Muscular Dystrophy is a desperately cruel muscle wasting disease that mainly affects boys. It’s the most common fatal genetic disease to affect children. It is fatal and boys die in their early 20s. There are limited treatment options and no cure.
Duchenne UK will be run by Emily Crossley, formerly 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, and will continue to run its own Joining Jack events.
Alex Johnson says: “Joining Jack will continue to run its own events. But 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 the Duchenne Research Fund in advocacy and research funding.
Kerry Rosenfeld from the Duchenne Research Fund says: “These are exciting times for research in Duchenne. I’m so proud to work closely with and complement Duchenne UK to help achieve our shared joint mission of curing Duchenne for all boys, 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 are both sit on the Medical Health and Research Authority’s (MHRA) Patient Group Consultative Forum. Alex Johnson is a trained patient advocate, having attended the Eurordis summer training school. She is a board member of United Parent Project Muscular Dystrophy.
They have recently appointed a Director of Research, Fiona Lawrence, to help drive their 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 patient with Duchenne muscular dystrophy together.”
For more information please visit our website: www.duchenneuk.org