The last day 24th International Annual Congress of the World Muscle Society (WMS) opened with a talk touching on a growing field in neuromuscular disorders – the use of digital […]
Many therapies being developed for Duchenne muscular dystrophy, including gene therapies, gene editing, and some exon-skipping approaches, rely on use of a virus to get the therapeutic components inside of […]
Even though it’s a conference of the World Muscle Society, the presentations on Thursday morning focused on the disease processes happening in parts of the body other than muscle. This […]
Today was the first full day of research talks and posters at the 24th International Annual Congress of the World Muscle Society (WMS). It’s a busy meeting, with nearly 1,000 […]
Greetings from Copenhagen, Denmark, where several members of the CureDuchenne team are attending the 24th International Annual Congress of the World Muscle Society (WMS). This is the largest annual international […]
Today’s FDA disapproval of Sarepta’s Vyondys 53 is disheartening, but a true example of just how difficult and costly the drug development process is. We appreciate Sarepta’s efforts and dedication […]
Pfizer made their first public disclosure on early data from their open-label Phase 1 microdystrophin gene therapy trial for the treatment of Duchenne. The program was obtained from Pfizer’s acquisition […]
We are excited to report the progress of CureDuchenne’s Gene Therapy Initiative. From our investment, as early as 2010, in Nationwide Children’s permanent duplication2 skipping program, to what has become […]
In 2003 when my son Hawken was diagnosed with Duchenne people were not contemplating a cure. To find a pharmaceutical company working on Duchenne was like finding a needle in […]
