Sarepta Therapeutics and Roche have announced that they will be working in partnership to develop Sarepta’s potential gene therapy for Duchenne …
Sarepta Therapeutics and Roche to partner on gene therapyRead More
Golodirsen gains conditional approval in US
Sarepta Therapeutics has announced that the FDA (the medicines regular in the US) has granted accelerated approval to golodirsen. Golodirsen, or …
Wave halts development of exon skipping drugs
Wave has today announced that they have discontinued the clinical program of suvodirsen (designed to skip exon 51 of the dystrophin gene) and are …
Welcome to our new Company Secretary
Action Duchenne is delighted to announce the appointment of Victoria Penrice as Company Secretary and trustee. Victoria is joining us at an exciting …
Jonathan raises the profile of Duchenne
Our amazing community have really got behind Jonathon Inkin singing his beautiful rendition of Jingle Bells. Jonathon is raising money for Action …
Duchenne Patient Academy in Athens
Last week our Trustee, Gary Fegan, was honoured to join delegates from Duchenne patient organisations from more than 40 countries worldwide …
Update on UNITE-DMD project
The UNITE-DMD project, which we are co-funding alongside Muscular Dystrophy UK and AFM-Téléthon brings together several strands of research to …
Duchenne Science events back on the road in 2020
We've welcomed over 100 people to our Science on Tour events throughout 2019, these empowering and invaluable sessions have been a huge success and we …
GA Solicitors – Charity of the Year
We were delighted to be chosen as Charity of the Year by Plymouth law firm GA Solicitors. As part of their fundraising the team organised …
A message from our Patron, Harry Hill
Harry Hill, one of our amazing Patrons, sent us a video which we proudly played at the Gala Dinner on Friday at the Action Duchenne International …
An unforgettable weekend at #ADCON19
Thank you to each and every person who attended the Action Duchenne International Conference 2019. We have all had such a wonderful weekend with …
Solid gene therapy trial placed on hold
Solid BioSciences has announced that the IGNITE DMD trial - a Phase I/II clinical trial of the company’s SGT-001 gene therapy - has been placed …
