Sarepta Therapeutics announced that the U.S. FDA has accepted the Company's Biologics License Application for SRP-9001 gene therapy for …
Sarepta Theraputics Gene Therapy Granted Priority Review by FDARead More
Support our new project this Christmas
Recently my son’s needs changed, his school was struggling and without the correct equipment and training in place a decision by the local authority …
Santhera and ReveraGen Complete NDA Submission to FDA for Vamorolone in Duchenne Muscular Dystrophy
Ad hoc announcement pursuant to Art. 53 LR Pratteln, Switzerland, and Rockville, MD, USA, October 27, 2022 – Santhera Pharmaceuticals (SIX: …
Important update for DMD parents and carers
Important update for DMD parents and carers:DMD Care UK’s new recommendations for cardiac care have been published in the BMJ Open Heart today.You can …
Peer-to-peer support – transitioning to secondary
This week's Friday Hive support session (as part of the All-through Support project) we talked about transitioning to secondary school and how to make …
NICE publishes draft guidance on access to Duchenne treatment Translarna
NICE has published draft guidance stating that although Translarna (also known as ataluren) is clinically effective and an innovative treatment, there …
NICE publishes draft guidance on access to Duchenne treatment TranslarnaRead More
Questions at the Conference
Action Duchenne is providing a platform for you, the Duchenne community, to ask questions from the leading Pharmaceutical companies driving …
Newly diagnosed family online event
Building your foundation of Duchenne knowledge Online via ZoomSaturday 22nd October 202214:00 - 17:00 GMTAll welcome More information An …
Conference agenda
Join us for two days in November as we explore the latest research, treatments and updates. Watch presentations, Q&A sessions and join discussions …
Edgewise announces FDA authorisation for Phase 2 clinical trial
Edgewise has announced the FDA has authorized Phase 2 clinical trial for the treatment of Duchenne. The Lynx trial is a placebo-controlled trial to …
Edgewise announces FDA authorisation for Phase 2 clinical trialRead More
Sarepta Therapeutics announces scholarship program
Twenty exceptional individuals living with Duchenne muscular dystrophy will receive a scholarship of up to $5,000 as they pursue their post-secondary …
PTC reveals winners of STRIVE awards on World Duchenne Awareness Day
Over the past few months, we are proud to have shared photos, updates and stories from our ‘Yes I can’ project. You’ll have seen the looks of …
PTC reveals winners of STRIVE awards on World Duchenne Awareness DayRead More
