Last week Action Duchenne attended the inaugural Rare Film Festival, hosted by Rare Disease UK. One of the sponsoring companies, PTC Therapeutics …
Give your heart to Bertie’s ball
It was an honour to attend the 'Give Your Heart to Bertie's Ball' organised by the wonderful Tracey Keilty and Kayleigh Coulson. Their commitment …
A warm welcome message from our new National Director
I am delighted to have been appointed as National Director of Action Duchenne. It is an honour to become part of a charity so dedicated to making a …
A warm welcome message from our new National DirectorRead More
Welcome to our new National Director
We are delighted to welcome Florence Boulton to Action Duchenne as our new National Director. Florence has considerable leadership and …
Take part in quality of life research for Duchenne
Acaster Lloyd, an independent research consultancy, are carrying out research looking at the impact of Duchenne on family carers, including people's …
Advice for Emergency Care
Children with DMD experience the normal childhood illnesses and accidents. But, because their muscles are weaker, some things are more serious for …
How many people with Duchenne might be eligible to receive idebenone in England?
In May 2019 Santhera Pharmaceuticals submitted an application for a European Marketing Authorisation for Puldysa® (idebenone) for the treatment of …
How many people with Duchenne might be eligible to receive idebenone in England?Read More
Sarepta Therapeutics and Roche to partner on gene therapy
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 …
