Dyne Therapeutics have reported their initial clinical data from the DELIVER trial of DYNE-251 in patients with Duchenne muscular dystrophy who are …
PepGen Announces First Patient Dosed in CONNECT1-EDO51 Phase 2 Clinical Trial of PGN-EDO51 for Duchenne Muscular Dystrophy Patients Amenable to Exon 51 Skipping
PepGen Inc., a clinical-stage biotechnology company advancing the next-generation of oligonucleotide therapies, today announced that the first patient …
Wave Life Sciences Announce the Start of the FORWARD-53 Clinical Study
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
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 Muscular Dystrophy
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 …
Dyne Therapeutics Receives FDA Orphan Drug and Rare Pediatric Designations for DYNE-251 for the Treatment of Duchenne Muscular Dystrophy
Dyne Therapeutics Receives FDA Orphan Drug and Rare Pediatric Designations for DYNE-251 for the Treatment of Duchenne Muscular Dystrophy Dyne …
Nationwide researchers announce restoration of full-length Dystrophin in humans
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
Dyne Therapeutics announce application to start exon 51 skipping clinical trial
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
Project that aimed to combine stem cell and CRISPR technology is a success
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
In just 7 weeks, we’ll be on the road again
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 …
Golodirsen gains conditional approval in US
Sarepta Therapeutics has announced that the FDA (the medicines regular in the US) has granted accelerated approval to golodirsen. Golodirsen, or …
Wave halts development of exon skipping drugs
Wave has today announced that they have discontinued the clinical program of suvodirsen (designed to skip exon 51 of the dystrophin gene) and are …
Duchenne Science events back on the road in 2020
We've welcomed over 100 people to our Science on Tour events throughout 2019, these empowering and invaluable sessions have been a huge success and we …
