- European Commission ratification anticipated in coming months - - Approval of the Translarna annual re-assessment also recommended by CHMP - PTC …
As Expected, Sarepta Receives Negative CHMP Opinion for EXONDYS (eteplirsen) to Treat Patients with Duchenne in Europe
Sarepta will seek re-examination of the opinion and request that a Scientific Advisory Group (SAG) be convened Sarepta have announced that the …
Daiichi Sankyo announces Phase 1/2 clinical trial results
Daiichi Sankyo have announced the top-line results of the Phase 1/2 clinical trial in Japan of DS-5141), which Daiichi Sankyo is jointly …
Daiichi Sankyo announces Phase 1/2 clinical trial resultsRead More
Support for the Shippey family at their annual Golf Day
96 amazing golfers and a further 34 evening guests gathered to support the wonderful Shippey family at their second annual Cure4George charity golf …
Support for the Shippey family at their annual Golf DayRead More
Sarepta collaborate with Invitae to speed up diagnosis of Duchenne
Muscular Dystrophy News Today have reported that Sarepta Therapeutics and Invitae, a company specialising in genetic diagnostics, are …
Sarepta collaborate with Invitae to speed up diagnosis of DuchenneRead More
Thomas Blumire’s music to be performed at West Midlands concert
Music of Life Foundation is a small charity that uses music-making to advance education and improve the health and wellbeing of disabled …
Thomas Blumire’s music to be performed at West Midlands concertRead More
FDA Grants Orphan Drug Designation to Sarconeos
BIOPHYTIS have announced that the US Food and Drug administration (FDA) has granted orphan drug designation to its drug candidate, Sarconeos. In …
Solid announces upcoming pre-clinical data presentations
Solid have announced that new pre-clinical data from its Duchenne programs, including its lead gene transfer candidate, SGT-001, will be …
Solid announces upcoming pre-clinical data presentationsRead More
Teachers brave the Zipwire!
This weekend saw an amazing group of teachers and supporters from Bruntcliffe Academy take on the world's fastest, and Europe's longest …
NICE update on Eteplirsen
Following last week’s announcement that Sarepta’s exon skipping drug, Eteplirsen, had received a negative trend vote from the …
Action Duchenne announces changes and new key appointments
At Action Duchenne we aim to do our utmost to communicate the many promising Duchenne and Becker Muscular Dystrophy research projects and …
Action Duchenne announces changes and new key appointmentsRead More
Catabasis financial reports and business progress
-- MoveDMD® Trial Data Through One Year of Treatment Reinforce Edasalonexent Potential as Disease-Modifying Therapy for Duchenne Muscular …
