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
Make a Difference – Join Our Charity Running Events!
Are your employees ready to lace up their running shoes and support a great cause? 🌟 We invite your team to join us at one of our upcoming …
Make a Difference – Join Our Charity Running Events!Read More
Do you live in Scotland or Wales? Join our Focus Groups to tell us what support YOU need!
Do you live in Scotland or Wales? Join our Focus Groups to tell us what support YOU need! Improving the lives of everyone that lives with DMD is …
Do you live in Scotland or Wales? Join our Focus Groups to tell us what support YOU need!Read More
