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
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Yes I Can In-Person Meet-Up at Think Tank, Birmingham
Yes I Can In-Person Meet-Up at Think Tank, Birmingham We are planning an in-person meet-up at Think Tank, the Science Museum in Birmingham. 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 Thomley On 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
Entrada Therapeutics Reports Positive Preliminary Data in Healthy Volunteers from Phase 1 ENTR-601-44-101 Trial for Duchenne Muscular Dystrophy
Entrada Therapeutics, a company developing treatments for rare diseases, reports encouraging preliminary data from a Phase 1 clinical trial …
Action Duchenne’s Science on Tour Team visit Ashford School
Action Duchenne Science on Tour Team visit Ashford School A new addition to this years’ Science on Tour is our school visits, part of our ongoing …
Action Duchenne’s Science on Tour Team visit Ashford SchoolRead More
