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 2Where 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 …
Wave gives suvodirsen update
Wave Life Sciences announced earlier this week that Suvodirsen has been granted fast track approval by the Federal Drug Administration (the drug …
Sarepta issues clinical trials update
In recent press releases, Sarepta Therapeutics has given updates on their ongoing clinical trial programmes. These include exon skipping and gene …
