PepGen, a biotech company developing innovative oligonucleotide therapies for severe neuromuscular and neurological diseases, has received approval …
PepGen Receives UK CTA Clearance from MHRA to Begin CONNECT2-EDO51 Phase 2 Clinical TrialRead More
This is the latest news about the development and use of exon skipping in Duchenne muscular dystrophy. There’s also lots of information and videos explaining these potential treatments in our information pages.
March 5, 2024 by John Marrin
PepGen, a biotech company developing innovative oligonucleotide therapies for severe neuromuscular and neurological diseases, has received approval …
PepGen Receives UK CTA Clearance from MHRA to Begin CONNECT2-EDO51 Phase 2 Clinical TrialRead More
January 31, 2024 by John Marrin
On the Monday the 29th of January, Sarepta Therapeutics announced positive data from Part B of the MOMENTUM study. The MOMENTUM Study is a global …
January 11, 2024 by John Marrin
Dyne Therapeutics have reported their initial clinical data from the DELIVER trial of DYNE-251 in patients with Duchenne muscular dystrophy who are …
January 11, 2024 by John Marrin
PepGen Inc., a clinical-stage biotechnology company advancing the next-generation of oligonucleotide therapies, today announced that the first patient …
December 21, 2023 by John Marrin
Wave Life Sciences Announce the Start of the FORWARD-53 Clinical Study On the 15th of December 2023, Wave Life Sciences announced the initiation …
<strong>Wave Life Sciences Announce the Start of the FORWARD-53 Clinical Study </strong>Read More
May 19, 2023 by Mehreen Arif
PepGen Announces Clearance by Health Canada of CTA for PGN-EDO51 to Begin the Phase 2 Clinical Trial, CONNECT1-EDO51, for the Treatment of Duchenne …
March 25, 2023 by Lizzie Deeble
Dyne Therapeutics Receives FDA Orphan Drug and Rare Pediatric Designations for DYNE-251 for the Treatment of Duchenne Muscular Dystrophy Dyne …
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
December 7, 2021 by Lynnette
Dyne Therapeutics, Inc. announced on 2nd December 2021 the submission of an Investigational New Drug (IND) application to the U.S. Food and Drug …
Dyne Therapeutics announce application to start exon 51 skipping clinical trialRead More
October 28, 2021 by Neil
We’re delighted to report the successful completion of our research project in Dr Yung-Yao Lin’s laboratory at Queen Mary University of London. The …
Project that aimed to combine stem cell and CRISPR technology is a successRead More
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 …
December 16, 2019 by Neil
Sarepta Therapeutics has announced that the FDA (the medicines regular in the US) has granted accelerated approval to golodirsen. Golodirsen, or …
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