If you are a caregiver of/or a young person aged 7-25 with Duchenne, researchers from the University of Glasgow, and Edge Hill University, need your …
Help Develop Nutrition Information Resources for Duchenne Muscular DystrophyRead More
Wave Life Sciences Announces Positive Interim Data from FORWARD-53 Clinical Trial Evaluating WVE-N531 in Boys with Duchenne Muscular Dystrophy Amenable to Exon 53 Skipping
Wave Life Sciences has shared their promising new interim results for the ongoing FORWARD-53 trial. The ongoing open-label trial, involves eleven boys …
Swissmedic Announces Acceptance of Santhera’s Market Authorisation Application for AGAMREE (Vamorolone) in Duchenne Muscular Dystrophy
Santhera Pharmaceuticals (SIX: SANN) has announced that Swissmedic, the Swiss Agency for Therapeutic Products, has begun reviewing the marketing …
Action Duchenne: Delivering Hope & Change in 2024
We are pleased to share our Impact 2024 report, which highlights our progress and achievements over the past year. It showcase the hard work and …
Together for World Duchenne Awareness Day: Celebrating Strength and Progress
"On World Duchenne Awareness Day 2024, we come together as a community—families, friends, carers, and supporters—alongside everyone living with …
Together for World Duchenne Awareness Day: Celebrating Strength and ProgressRead More
Help Improve Understanding of Nutrition and Weight Management in DMD
Do you have 15 minutes and want to help improve our scientific understanding of the influence of nutrition and weight management in Duchenne muscular …
Help Improve Understanding of Nutrition and Weight Management in DMDRead More
The Action Duchenne Conference was a game-changer for me as a new Duchenne dad.
The Action Duchenne Conference was a game-changer for me as a new Duchenne dad. It gave me the knowledge I needed through clear talks on treatment and …
The Action Duchenne Conference was a game-changer for me as a new Duchenne dad.Read More
Clinical Trial Update – PepGen Announces Positive Data from Ongoing CONNECT1-EDO51 Phase 2 Trial for Treatment of Duchenne Muscular Dystrophy
PepGen Inc., announced on July 30th 2024, positive clinical data from the first dose cohort (5 mg/kg) of PGN-EDO51, its lead investigational candidate …
Clinical Trial Update – RegenxBio Announces New Positive Data From Affinity Duchenne Trial of RGX-202
On the 1st of August, RiogenxBio provided a positive update on the interim safety and efficacy data collected from their Phase I/II AFFINITY DUCHENNE …
Important Update on Pfizer’s CIFFREO Phase 3 Clinical Trial for DMD Gene Therapy
In June, Pifzer announced that their CIFFREO Phase 3 clinical trial for Duchenne muscular dystrophy (DMD) gene therapy did not meet its primary and …
Important Update on Pfizer’s CIFFREO Phase 3 Clinical Trial for DMD Gene TherapyRead More
CEO’s Message: Welcoming New Trustees and Farewell to Victoria Penrice
CEO's Message: Welcoming New Trustees and Farewell to Victoria Penrice Action Duchenne is delighted to announce the appointment of three …
CEO’s Message: Welcoming New Trustees and Farewell to Victoria PenriceRead More
