We are delighted to share the news with our community that the award-winning Demelza Children's Hospice have chosen us as their partners to deliver an …
Action Duchenne receives funding for ground-breaking transition projectRead More
Q&A on the Managed Access Agreement (MAA) for Translarna/ataluren in England
Translarna/ataluren is used to treat Duchenne muscular dystrophy that is caused by nonsense mutations. Translarna is currently accessed in England …
Q&A on the Managed Access Agreement (MAA) for Translarna/ataluren in EnglandRead More
Update on Translarna NHS treatment for Duchenne muscular dystrophy
NICE has today announced that the Managed Access Agreement (MAA) for the drug Translarna has been extended until January 2023. The extension means …
Update on Translarna NHS treatment for Duchenne muscular dystrophyRead More
World Duchenne Awareness Day 2021
September 7 is World Duchenne Awareness Day. On this day we join the international Duchenne community to raise the profile and awareness of the …
Alex James release new single
The phenomenal North-East based alternative indie-rock band, Alex James, are set to make an unprecedented return, following the success of their debut …
In just 7 weeks, we’ll be on the road again
Duchenne Science on Tour 2 Where we'll be helping our families to make informed choices around really complex things like gene therapy and exon …
Translarna accepted by Scottish Medicines Consortium
A drug called Translarna can help treat an underlying condition of Duchenne muscular dystrophyAction Duchenne, Duchenne Family Support Group, Muscular …
Translarna accepted by Scottish Medicines ConsortiumRead More
Public statement from NICE
Action Duchenne and MDUK have received the following public statement from NICE which we can share with the community: "The Managed Access …
The Spires Federation school fundraising
The awesome Deborah Holland is taking on the mighty Peak District Challenge in July, inspired by her best friend Lindsay's youngest son Riley who …
Rare disease day
Rare diseases present unique challenges. Many families have never heard of Duchenne before a diagnosis. It might be the first case your GP has …
A message from Florence Boulton on Rare Disease Day 2021
Dear International Duchenne Community, Today, we take a moment from our daily lives to raise awareness about our community and share what it means …
A message from Florence Boulton on Rare Disease Day 2021Read More
Happy birthday John Miller
Dedicated Grandfather and our Scottish Advocate John Miller has selflessly created a birthday fundraiser for Action Duchenne to celebrate his 82nd …
