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  • About Us
    • Our Purpose
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    • Volunteer
    • The DMD Registry
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  • Get Support
    • Support Calendar – What’s On
    • Register for Support
      • Time Out – A Space for Mums
      • Dads Against Duchenne
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    • Recently diagnosed
    • Children and Young People
    • In-Person Support Events
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    • Highlights from the Action Duchenne Conference 2025
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    • Webinar recordings
    • Bite-Sized Duchenne Science Live
      • Facts about Duchenne muscular dystrophy
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      • Diagnosis of Duchenne Muscular Dystrophy
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      • Genetics – Blueprint of Duchenne Muscular Dystrophy
      • How is Duchenne Muscular Dystrophy Inherited?
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News

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Swissmedic Announces Acceptance of Santhera’s Market Authorisation Application for AGAMREE (Vamorolone) in Duchenne Muscular Dystrophy

11 October 2024 by John Marrin

Santhera Pharmaceuticals (SIX: SANN) has announced that Swissmedic, the Swiss Agency for Therapeutic Products, has begun reviewing the marketing …

Swissmedic Announces Acceptance of Santhera’s Market Authorisation Application for AGAMREE (Vamorolone) in Duchenne Muscular DystrophyRead More

Action Duchenne: Delivering Hope & Change in 2024

27 September 2024 by Lizzie Cox

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 …

Action Duchenne: Delivering Hope & Change in 2024Read More

Together for World Duchenne Awareness Day: Celebrating Strength and Progress

6 September 2024 by Lizzie Cox

"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

28 August 2024 by John Marrin

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.

21 August 2024 by Lizzie Cox

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

5 August 2024 by John Marrin

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 – PepGen Announces Positive Data from Ongoing CONNECT1-EDO51 Phase 2 Trial for Treatment of Duchenne Muscular DystrophyRead More

Clinical Trial Update – RegenxBio Announces New Positive Data From Affinity Duchenne Trial of RGX-202

5 August 2024 by John Marrin

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 …

Clinical Trial Update – RegenxBio Announces New Positive Data From Affinity Duchenne Trial of RGX-202Read More

Important Update on Pfizer’s CIFFREO Phase 3 Clinical Trial for DMD Gene Therapy

31 July 2024 by Victoria Edwards

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

10 July 2024 by Lizzie Cox

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

Submit Your Questions to be Answered by Pharmaceutical Companies

9 July 2024 by John Marrin

…

Submit Your Questions to be Answered by Pharmaceutical CompaniesRead More

Yes I Can In-Person Meet-Up at Think Tank, Birmingham

6 July 2024 by Lizzie Cox

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

5 July 2024 by John Marrin

PTC Therapeutics has announced that the European Medicine Agency (EMA) Committee for Medicinal Products for Human Use (CHMP) issued a negative …

The Committee for Medicinal Products for Human Use (CHMP) Issues Negative Opinion on Translarna™ Following European Commission Request for ReviewRead More

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