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
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
Spotlight on fundraisers January 2018
Jo Gelblum is a serial fundraiser Grandparent who raised £1,984 selling cakes over Christmas. Members of the Marks Tey Silver Threads …
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 …
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 …
Action Duchenne raise over £30,000 this year at charity ballsRead More
The importance of seeking medical advice – Neuromuscular Research Nurse
The use of social media parent groups is invaluable for parents whose children have DMD. They provide a great source of emotional and practical …
The importance of seeking medical advice – Neuromuscular Research NurseRead More
FDA new approach would reduce number of patients treated with placebo
The U.S. Food and Drug Administration (FDA) has published its recommendation for a new approach to drug development for rare pediatric diseases. The …
FDA new approach would reduce number of patients treated with placeboRead More
Mark Silverman is poised to deliver his powerful Translarna testimonial to the FDA
"I was last in Washington DC two years ago and let's just say there have been a few changes since my last visit. The searing late September heat …
Mark Silverman is poised to deliver his powerful Translarna testimonial to the FDARead More
Acceleron takes back drug rights from Celgene to start pulmonary push
Acceleron's long-running collaboration with Celgene on sotatercept has taken another turn, with an amendment of their agreement to give Acceleron the …
Acceleron takes back drug rights from Celgene to start pulmonary pushRead More
Synpromics announces Gene Therapy research partnership with Solid Biosciences
Sypromics have announced a new research partnership with Solid Biosciences. Under the terms of the agreement, Synpromics will provide Solid …
Synpromics announces Gene Therapy research partnership with Solid BiosciencesRead More
