• About Us
    • Our Purpose
    • Our Strategy
    • What we do
    • Our Impact
    • Our team
    • Work For Us
    • Volunteer
    • The DMD Registry
    • Action Duchenne Policies
  • Get Support
    • Support Calendar – What’s On
    • In-Person Support Events
    • Register for Support
      • Time Out – A Space for Mums
      • Dads Against Duchenne
      • Grandparents Together
      • Open Space
      • Group Counselling Programme
    • Recently diagnosed
    • Schools
    • Siblings
    • End of Life and Bereavement
  • AD Annual International Conference
    • Highlights from the Action Duchenne Conference 2025
    • Action Duchenne Annual International Conference 2025
  • News, Webinars and Blogs
    • News
    • Webinar Series 2026
    • Webinar recordings
    • Bite-Sized Duchenne Science Live
      • Facts about Duchenne muscular dystrophy
      • Signs and Symptoms of Duchenne Muscular Dystrophy
      • Diagnosis of Duchenne Muscular Dystrophy
      • Crucial Genetic Terminology
      • Genetics – Blueprint of Duchenne Muscular Dystrophy
      • How is Duchenne Muscular Dystrophy Inherited?
    • Hear From Our Community
  • Support Us
  •  0 items - £0.00
  • Menu
  • Skip to right header navigation
  • Skip to main content
  • Skip to secondary navigation
  • Skip to footer

Before Header

  • My account
  •  0 items - £0.00

Action Duchenne

Header Right

  • About Us
    • Our Purpose
    • Our Strategy
    • What we do
    • Our Impact
    • Our team
    • Work For Us
    • Volunteer
    • The DMD Registry
    • Action Duchenne Policies
  • Get Support
    • Support Calendar – What’s On
    • In-Person Support Events
    • Register for Support
      • Time Out – A Space for Mums
      • Dads Against Duchenne
      • Grandparents Together
      • Open Space
      • Group Counselling Programme
    • Recently diagnosed
    • Schools
    • Siblings
    • End of Life and Bereavement
  • AD Annual International Conference
    • Highlights from the Action Duchenne Conference 2025
    • Action Duchenne Annual International Conference 2025
  • News, Webinars and Blogs
    • News
    • Webinar Series 2026
    • Webinar recordings
    • Bite-Sized Duchenne Science Live
      • Facts about Duchenne muscular dystrophy
      • Signs and Symptoms of Duchenne Muscular Dystrophy
      • Diagnosis of Duchenne Muscular Dystrophy
      • Crucial Genetic Terminology
      • Genetics – Blueprint of Duchenne Muscular Dystrophy
      • How is Duchenne Muscular Dystrophy Inherited?
    • Hear From Our Community
  • Support Us

Gene therapy

You are here: Home / News / Gene therapy

Sarepta Therapeutics Announces FDA Approval of ELEVIDYS, the First Gene Therapy to Treat Duchenne Muscular Dystrophy

June 23, 2023 by Mehreen Arif

Sarepta Therapeutics announced U.S. Food and Drug Administration (FDA) accelerated approval of ELEVIDYS (delandistrogene …

Sarepta Therapeutics Announces FDA Approval of ELEVIDYS, the First Gene Therapy to Treat Duchenne Muscular DystrophyRead More

Roche UK issues statement to the Duchenne Community following Sarepta’s Update on Regulatory Review of SRP-9001 gene therapy.

May 25, 2023 by Mehreen Arif

Roche UK issues statement to the Duchenne Community following Sarepta's Update on Regulatory Review of SRP-9001 gene therapy. Following the update …

Roche UK issues statement to the Duchenne Community following Sarepta’s Update on Regulatory Review of SRP-9001 gene therapy.Read More

Sarepta Therapeutics Announces Update on Regulatory Review of SRP-9001

May 24, 2023 by Mehreen Arif

Sarepta Therapeutics Announces Update on Regulatory Review of SRP-9001 Sarepta Therapeutics, today provided the following update on the Biologics …

Sarepta Therapeutics Announces Update on Regulatory Review of SRP-9001Read More

Sarepta Therapeutics Announces Positive Vote from U.S. FDA Advisory Committee Meeting for SRP-9001 Gene Therapy to Treat Duchenne Muscular Dystrophy

May 17, 2023 by Mehreen Arif

Sarepta Therapeutics Announces Positive Vote from U.S. FDA Advisory Committee Meeting for SRP-9001 Gene Therapy to Treat Duchenne Muscular …

Sarepta Therapeutics Announces Positive Vote from U.S. FDA Advisory Committee Meeting for SRP-9001 Gene Therapy to Treat Duchenne Muscular DystrophyRead More

Sarepta Theraputics Gene Therapy Granted Priority Review by FDA

December 5, 2022 by Lizzie Cox

Sarepta Therapeutics announced that the U.S. FDA has accepted the Company's Biologics License Application for SRP-9001 gene therapy for …

Sarepta Theraputics Gene Therapy Granted Priority Review by FDARead More

Pfizer shares update on Phase 2 DAYLIGHT clinical trial

September 6, 2022 by Lynnette

We are pleased to share an update with you from US based pharmaceutical and biotechnology corporation, Pfizer about their clinical …

Pfizer shares update on Phase 2 DAYLIGHT clinical trialRead More

Nationwide researchers announce restoration of full-length Dystrophin in humans

May 27, 2022 by Mehreen Arif

In Duchenne muscular dystrophy, dystrophin protein is absent or partially functional due to mutations in the dystrophin gene. Multiple therapeutic …

Nationwide researchers announce restoration of full-length Dystrophin in humansRead More

Pfizer to re-start its global Phase 3 Trial of Investigational Gene Therapy for Ambulatory Patients with Duchenne Muscular Dystrophy

May 5, 2022 by Lynnette

 Pfizer has announced on 28 April 2022 they have received approvals to re-start the Phase 3 study evaluating their gene therapy for Duchenne muscular …

Pfizer to re-start its global Phase 3 Trial of Investigational Gene Therapy for Ambulatory Patients with Duchenne Muscular DystrophyRead More

Gene therapy trial shows ‘statistically significant’ improvements

January 27, 2022 by Victoria Edwards

Sarepta Therapeutics have announced the audited, quality-controlled data reflecting all results from Part 2 of their Study SRP-9001-102 (Study 102). …

Gene therapy trial shows ‘statistically significant’ improvementsRead More

In just 7 weeks, we’ll be on the road again

May 4, 2021 by Samantha

Duchenne Science on Tour 2 Where we'll be helping our families to make informed choices around really complex things like gene therapy and exon …

In just 7 weeks, we’ll be on the road againRead More

Gene therapy experts announced

September 17, 2020 by Lynnette

Have we got an exciting line up of speakers and sessions for you! We will be announcing more details over the coming days and weeks. But for …

Gene therapy experts announcedRead More

Latest results from Solid gene therapy trial

March 12, 2020 by Neil

Solid Biosciences have today released an update on their clinical trial of a gene therapy for Duchenne.  The IGNITE DMD trial is an early …

Latest results from Solid gene therapy trialRead More

  • « Go to Previous Page
  • Page 1
  • Page 2
  • Page 3
  • Go to Next Page »

Footer

Action Duchenne
5th Floor, Mariner House
62 Prince Street
Bristol
BS1 4QD

07535 498 506
info@actionduchenne.org 

 

 

 

 

 

 

 

 

Subscribe to our mailing list

Do you consent to receiving regular email updates? *
Email Format
  • Accessibility
  • Privacy Policy
  • Terms & Conditions

© Action Duchenne - Registered Charity No 1101971 - Scottish Charity No SC043852

Like most websites we use cookies to deliver a personalised service. To use the website as intended please accept cookies.
Privacy & Cookies Policy

Privacy Overview

This website uses cookies to improve your experience while you navigate through the website. Out of these, the cookies that are categorized as necessary are stored on your browser as they are essential for the working of basic functionalities of the website. We also use third-party cookies that help us analyze and understand how you use this website. These cookies will be stored in your browser only with your consent. You also have the option to opt-out of these cookies. But opting out of some of these cookies may affect your browsing experience.
Necessary
Always Enabled
Necessary cookies are absolutely essential for the website to function properly. This category only includes cookies that ensures basic functionalities and security features of the website. These cookies do not store any personal information.
Non-necessary
Any cookies that may not be particularly necessary for the website to function and is used specifically to collect user personal data via analytics, ads, other embedded contents are termed as non-necessary cookies. It is mandatory to procure user consent prior to running these cookies on your website.
SAVE & ACCEPT