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 …
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
Neil test news story
Newly diagnosed families Newly diagnosed families …
PerkinElmer team up with IDG in diagnostic program
PerkinElmer have announced a collaboration with In-Depth Genomics (IDG) to support IDG's Whole Genome Sequencing (WGS) Diagnostic Program that will …
