Wales has joined the 100,000 Genomes Project, the Welsh Government announced. The project, now a UK-wide initiative, seeks to transform patient …
PTC receives formal dispute resolution request decision from the FDA
PTC have today announced that the Office of New Drugs of the U.S. Food and Drug Administration has reiterated the FDA's prior …
PTC receives formal dispute resolution request decision from the FDARead More
FDA share guidance for Duchenne and Becker muscular dystrophy drug development
We are happy to share the news that the U.S. Food and Drug Administration (FDA) have published a guidance document for industry, which details what …
FDA share guidance for Duchenne and Becker muscular dystrophy drug developmentRead More
Summit discuss their recently reported positive data from their ‘PhaseOut DMD’ trial (webinar)
Download the FAQ Bringing you the most up to date news from the international Duchenne community is one of our core aims. Action Duchenne and …
Catabasis reports Edsalonexent preserved muscle function and slowed progression of Duchenne through more than one year of treatment
Catabasis have reported new positive efficacy and safety results showing preservation of muscle function and sustained condition-modifying effects in …
Capricor receives FDA RMAT designation for CAP-1002
Capricor have announced that the U.S. Food and Drug Administration (FDA) has granted CAP-1002 the Regenerative Medicine Advanced Therapy (RMAT) …
Capricor receives FDA RMAT designation for CAP-1002Read More
Investigational idebenone becomes available through EAP in US
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
