Sarepta therapeutics has today announced that they have completed the submission of a USA application for accelerated approval of golodirsen …
Sarepta completes accelerated approval application for golodirsenRead More
UNITE-DMD project update – Year 1
Gene therapy for Duchenne muscular dystrophy aims to compensate for the lack of dystrophin by transferring a working version of the dystrophin gene …
Wave Life Sciences announce phase 1 trial results
In a press release published earlier, Wave Life Sciences has announced positive results of the phase 1 trial of the WVE-210201 – a potential …
Harry Hill’s ‘Comedians Sing Christmas Songs’ for Action Duchenne was a hit!
A huge thank you to our patron, Harry Hill, who hosted one of Action Duchenne's biggest ever events last night. 'Comedians Sing The Christmas Hits' …
Harry Hill’s ‘Comedians Sing Christmas Songs’ for Action Duchenne was a hit!Read More
ENMC Workshop in Amsterdam
ENMC has a unique concept of facilitating and organizing workshops for experts in the field of neuromuscular conditions. Every month a group of 20 - …
Action Duchenne present parent’s perspective to healthcare professionals
Last Friday, our Community Fundraising & Marketing Officer, Lynnette, was asked to present Duchenne from a parent's perspective to a number of …
Action Duchenne present parent’s perspective to healthcare professionalsRead More
Santhera acquires rights to vamorolone
Santhera has announced that they have acquired the rights to vamorolone - a potential drug that has been developed and is currently being tested in …
Comedians sing the Christmas hits!
It's gonna be one unholy night of fun! We are delighted to announce that on 4th December 2018, our patron Harry Hill will be hosting a comedy …
WE NEED YOUR VOTE – Chelsea vs Fulham matchday collection
Help us to win the chance to hold a bucket collection at Stamford Bridge at the Chelsea vs Fulham game Sunday 2 December. WE NEED YOUR VOTES - …
WE NEED YOUR VOTE – Chelsea vs Fulham matchday collectionRead More
Sarepta Therapeutics has announced the latest results from their gene therapy trial
Sarepta therapeutics has announced a further set of preliminary results of a gene therapy trial that is using a harmless virus to deliver a small but …
Sarepta Therapeutics has announced the latest results from their gene therapy trialRead More
Catabasis starts Phase 3 trial of edasalonexent
Earlier this week Catabasis announced that a Phase 3 trial of edasalonexent in Duchenne has started, with trial centres to open shortly – first …
Sarepta Therapeutics gene therapy trial to continue
Sarpeta Therapeutics has announced that the Food and Drug Administration (FDA – the drug regulator in the USA) has lifted the Clinical Hold that was …
Sarepta Therapeutics gene therapy trial to continueRead More
