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 …
Results from Italfarmaco’s EPIDYS trial
Italfarmaco Group announces positive topline data from Phase 3 trial showing beneficial effect of Givinostat in patients with Duchenne muscular …
Indoor Skydiving Experience Giveaway!
*Competition now closed* A fantastic experience worth £67.99! The winner receives the equivalent free-fall time of 3 real skydives, flight …
Edgewise Therapeutics announces positive 2-month interim results from the ARCH open label study of EDG-5506 in adults with Becker muscular dystrophy (BMD)
This article summaries the 2-month interim results shared by Edgewise Therapeutics from the ARCH study, evaluating EDG-5506 in adults with Becker …
Register for ‘Yes I can’ online and residential
'Yes I can' online workshops Every fortnight, on a Wednesday at 7pm, Ravi will be joined online by expert speakers, covering a range of important …
