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 …
Update on CHMP negative opinion for Raxone (idebenone)
This afternoon, Action Duchenne has since had confirmation by the Santhera team and this article that this decision has no consequences on their …
Update on CHMP negative opinion for Raxone (idebenone)Read More
First person reaches 15 month mark through Phrixus’ Carmseal-MD
Phrixus Pharmaceuticals has announced that the first person living with Duchenne has completed 15 months of treatment with Carmeseal-MD. The …
First person reaches 15 month mark through Phrixus’ Carmseal-MDRead 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). …
Parallel 2017 was amazing!
Parallel London 2017 was an absolutely inspirational day! 60 incredible people took part for Action Duchenne, representing the entire community, …
Sarconeos announced as new potential treatment for Duchenne
Biophytis clinical stage drug-candidate Sarconeos demonstrates efficacy in preclinical models of Duchenne muscular dystrophy An abstract …
Sarconeos announced as new potential treatment for DuchenneRead 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. Janet Bloor, Chair of …
Breaking news – first people enrolled in Raxone EAMS scheme in UKRead More
