"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 PenriceAction Duchenne is delighted to announce the appointment of three distinguished …
CEO’s Message: Welcoming New Trustees and Farewell to Victoria PenriceRead More
Submit Your Questions to be Answered by Pharmaceutical Companies
Yes I Can In-Person Meet-Up at Think Tank, Birmingham
Yes I Can In-Person Meet-Up at Think Tank, BirminghamWe are planning an in-person meet-up at Think Tank, the Science Museum in Birmingham. As well as …
Yes I Can In-Person Meet-Up at Think Tank, BirminghamRead More
The Committee for Medicinal Products for Human Use (CHMP) Issues Negative Opinion on Translarna™ Following European Commission Request for Review
PTC Therapeutics has announced that the European Medicine Agency (EMA) Committee for Medicinal Products for Human Use (CHMP) issued a negative …
Turning Point Families Day at Thomley
Turning Point Families Day at ThomleyOn Saturday 22nd June Dougie and I met up with other families at Thomley for the TurningPoint families …
A day to remember for Duchenne families with Alder Hey Children’s Hospital
On Thursday May 30th, we supported Alder Hey Children's Hospital hosting a special event for families affected by Duchenne muscular dystrophy (DMD). …
A day to remember for Duchenne families with Alder Hey Children’s HospitalRead More
