Catabasis reports Edsalonexent preserved muscle function and slowed progression of Duchenne through more than one year of treatment

Consistent improvements sustained across all assessments of muscle function through 48 and 60 weeks of Edasalonexent treatment in MoveDMD® trial. Single global Phase 3 trial expected to begin in first half of 2018.Read more

Investigational idebenone becomes available through EAP in US

Through the BreatheDMD program, people with Duchenne can obtain access to investigational idebenone, at no cost, through a growing network of research centres across the U.S.Read more

Capricor receives FDA RMAT designation for CAP-1002

CAP-1002 has been shown to exert potent immunomodulatory activity and stimulate cellular regenerationRead more

TREAT-NMD steroids analysis demonstrates the power of 'real-world' Registry data

The data may help to power future clinical trials by highlighting the need for trial design to take into account the heterogeneity of the DMD population including patient age, mutation type, and corticosteroid background.Read more

Implementation plans for the UK Strategy for Rare Diseases

Action Duchenne is delighted to hear the continued commitment by NHS England to improve care of those living with rare diseases. This coincides nicely with the release of the updated standard of care guidelines for Duchenne just last week.Read more

Santhera Receives Negative CHMP Opinion on Appeal for Authorization of Raxone® in Duchenne

Santhera Pharmaceuticals announces that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) maintained its negative opinion on the Type II extension application for Raxone® (idebenone) in Duchenne.Read more

Revised Standards of Care for Duchenne muscular dystrophy

Action Duchenne originally co-funded the translation of the original standard of care guidelines, into the widely used family guide and continues to be at the forefront of these developments.Read more

Positive data from Summit's PhaseOUT DMD ezutromid clinical trial

Summit have announced significant reduction in muscle damage and an increase in utrophin in muscle biopsies. Register for WEBINAR.Read more

Press release: Action Duchenne launches new Research Strategy

The new research strategy will focus on a number of areas of unmet need, including making access to trials possible for the entire Duchenne population (both paediatric and adult); many of the challenges for teenagers and adults living with Duchenne remain largely unaddressed:Read more

Give your valuable perspective in an anxiety study for young people and families living with Duchenne

Dubowitz Neuromuscular Centre and UCL Great Ormond Street Institute of Child Health hope this study will provide valuable information for caregivers and care providers, which will help to improve the support for people with Duchenne who are affected by anxiety.Read more

Families gain knowledge and support at Wales regional roundtable meeting

On Saturday 20 January 2018, Action Duchenne hosted a Wales round table meeting in Llandough Hospital. It gave Welsh families the opportunity to get a better understanding of the future outlook for those living with Duchenne in Wales and to see first-hand progress being made which could make major differences in improving the quality of life for those living with Duchenne.Read more

First patient dosed in microdystrophin gene therapy in US

"Action Duchenne are delighted to see the progress in gene therapy...originally identified gene therapy as it's top priority in 2013 and are continuing to fund this progress with the UNITE-DMD project with other fellow charities."Read more

Nominations open for Muscle Dream Rugby Experience

Join the Muscle Help Foundation at Twickenham Stadium for the England v Ireland 2-day Muscle Dream Experience.Read more

Action Duchenne raise over £30,000 this year at charity balls

Lesley Wegg, the Maurice family and the Ward family have all held charity balls for Action Duchenne this year raising an amazing total of £30,000 between them.Read more

The importance of seeking medical advice - Neuromuscular Research Nurse

Although parents will have a lot of experience when their own child is unwell or advice on what medication regime they feel works best for their child, it is essential that for all medical related queries or problems that a health professional is approached.Read more

FDA new approach would reduce number of patients treated with placebo

Through the proposed controlled, multi-arm, multi-company clinical trials, several products can be tested in a more time-efficient manner.Read more

Update on CHMP negative opinion for Raxone (idebenone)

the European Medicines Agency’s (EMA) decision making processes, adopted a negative opinion, recommending the refusal of a change to the marketing authorisation for the medicinal product Raxone.Read more

First person reaches 15 month mark through Phrixus' Carmseal-MD

First patient to be treated with Carmeseal-MD (P-188 NF) outside of the United States through Phrixus’s Expanded Access Program with Ethicor Pharma LtdRead more

Parallel 2017 was amazing!

60 incredible people took part for Action Duchenne, representing the entire community, along with the scores of supporters who were cheering on the Action Duchenne Parallel Heroes.Read more

Long-term microdystrophin pre-clinical work, moving towards a potential therapy restores muscle function in canines

Promising microdystrophin pre-clinical work, moving towards a potential therapy has shown to restore muscle function in the canine model (dogs).Read more

Sarconeos announced as new potential treatment for Duchenne

Biophytis clinical stage drug-candidate Sarconeos demonstrates efficacy in preclinical models of Duchenne muscular dystrophyRead more

Breaking news - first people enrolled in Raxone EAMS scheme in UK

We are delighted to announce the first people are enrolled in the UK's Early Access to Medicines Scheme (EAMS) for Raxone.Read more

Action Duchenne are Delighted to Announce Our New Campaigns Officer: Kathy

Mum to Isaac, who is living with Duchenne muscular dystrophy, I’ve been an active campaigner with Action Duchenne since 2011. Having found the support of the Duchenne community at the Action Duchenne conference in 2008, several months after Isaac was diagnosed, the annual conference helped my husband Stuart and me through the next couple of years as we reshaped our lives with the reality of Duchenne.Read more