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
Sarepta updates on MOMENTUM clinical trial
Sarepta Therapeutics has announced that the FDA has lifted its clinical hold on SRP-5051 (vesleteplirsen). Designed to skip exon 51 of the …
Pfizer shares update on Phase 2 DAYLIGHT clinical trial
We are pleased to share an update with you from US based pharmaceutical and biotechnology corporation, Pfizer about their clinical …
Pfizer shares update on Phase 2 DAYLIGHT clinical trialRead More
Action Duchenne International Conference
‘Educating, enabling and including’ is the theme of the unmissable 20th Action Duchenne International Conference, which launches this week. Bringing …
PTC transition survey
We are working with PTC Therapeutics to get answers to important questions about transition from paediatric to adult care. PTC would like to …
Sarepta recruiting for MIS51ON exon skipping study
Sarepta has announced they are recruiting for their MIS51ON clinical research study in the UK. A randomized, double-blind, dose finding and …
Translarna update
The treatment ataluren (Translarna) for Duchenne muscular dystrophy has now exited managed access and entered the NICE re-evaluation process. In order …
Step by step guide to the EHCP process
In this session Clair Warner, SEATTS PD Team Manager, shares her decades of specialist knowledge and experience to help our families navigate the …
Niall O’Doherty’s birthday fundraiser
Do you want to join me in making a difference? I'm raising money in aid of Action Duchenne and every donation will help. Thank you in advance for your …
