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  • Challenge 79 for World Duchenne Awareness Day
  • About Us
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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 Young

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

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