Santhera announces the launch of a U.S. Expanded Access Program (EAP) referred to as BreatheDMD with idebenone for patients with DMD. Through the …
Investigational idebenone becomes available through EAP in USRead More
TREAT-NMD steroids analysis demonstrates the power of ‘real-world’ Registry data
This study underlines the importance of data collection via patient registries and the critical role of multicenter collaboration in the rare disease …
TREAT-NMD steroids analysis demonstrates the power of ‘real-world’ Registry dataRead 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 …
Implementation plans for the UK Strategy for Rare DiseasesRead More
Revised Standards of Care for Duchenne muscular dystrophy
We are delighted to announce that the revised Standards of Care for Duchenne were published in The Lancet Neurology this week; a high-impact …
Revised Standards of Care for Duchenne muscular dystrophyRead More
Santhera Receives Negative CHMP Opinion on Appeal for Authorization of Raxone® in Duchenne
On 14 September 2017, the CHMP of the European Medicines Agency (EMA) adopted a negative opinion, recommending the refusal of a change to the …
Santhera Receives Negative CHMP Opinion on Appeal for Authorization of Raxone® in DuchenneRead More
Breaking news – updated Standards of Care published
Diagnosis and management of Duchenne muscular dystrophy Part 1: diagnosis, and neuromuscular, rehabilitation, endocrine, and gastrointestinal and …
Breaking news – updated Standards of Care publishedRead More
Positive data from Summit’s PhaseOUT DMD ezutromid clinical trial
Increase in utrophin protein expression observed Summit accelerating preparations for pivotal clinical trial Ezutromid is a potential …
Positive data from Summit’s PhaseOUT DMD ezutromid clinical trialRead More
Action Duchenne launches new Research Strategy
Action Duchenne, a leading UK-wide patient and parent-led organisation for Duchenne Muscular Dystrophy, is delighted to announce the publication of a …
Anxiety study for young people and families living with Duchenne
The Dubowitz Neuromuscular Centre, UCL Great Ormond Street Institute of Child Health are conducting a study which will look at anxiety in Duchenne …
Anxiety study for young people and families living with DuchenneRead More
Families gain knowledge and support at Wales regional roundtable meeting
It was useful to understand the services available for adults in Cardiff as well as an update on future treatments. Action Duchenne have provided a …
Families gain knowledge and support at Wales regional roundtable meetingRead More
First patient dosed in microdystrophin gene therapy in US
Parent Project Muscular Dystrophy (PPMD) announced that the first patient has been dosed with microdystrophin gene therapy by Dr. Jerry Mendell, …
First patient dosed in microdystrophin gene therapy in USRead 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. This fully hosted VIP programme is …
