Last week our incoming CEO, Shelley Simmonds, completed a 5 day intensive training programme in Barcelona, having been selected to take part in the …
Sarepta announce early, preliminary gene therapy trial results
Sarepta therapeutics has announced preliminary results of a gene therapy trial that is using a harmless virus to deliver a small but functional …
Sarepta announce early, preliminary gene therapy trial resultsRead More
DMD Pathfinders launch new nutrition guide for adults
DMD Pathfinders have worked with global health professionals to create a guide which provides information on nutrition for adults with Duchenne. They …
DMD Pathfinders launch new nutrition guide for adultsRead More
Solid Biosciences announce FDA have removed clinical hold from their gene therapy trial
Solid Biosciences have announced that the FDA has given approval for the company to restart the phase I/II IGNITE DMD clinical trial. The clinical …
Solid Biosciences announce FDA have removed clinical hold from their gene therapy trialRead More
Career Legal Bake Off is a great success!
Thank you to the wonderful Abbie Smith and Michelle McDermott of Career Legal for organising the fantastic Bake Off on Friday 8 June for Action …
New technology could highlight potential treatments for Duchenne muscular dystrophy
Researchers from Queen Mary University of London have developed new cell-based technologies which could improve understanding of the muscle-wasting …
New technology could highlight potential treatments for Duchenne muscular dystrophyRead More
Action Duchenne represent our community at bone protective therapy ENMC workshop
Bone Protective Therapy in Duchenne MD Date: 2018-06-03 Number: 236 City: Hoofddorp, The Netherlands Description: 236th ENMC …
Action Duchenne represent our community at bone protective therapy ENMC workshopRead More
Take part in ventilator study and earn £75
SuAzio are currently conducting a study and are looking for individuals who live with Duchenne, or their caregivers to be part of this study. …
CHMP Adopts Positive Opinion for the Expansion of the Translarna (ataluren) Label to Include Patients as Young as 2 Years of Age
- 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
