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 biomarkers to understand disease progression and whether interventions are effective.  This can include use of elaborate full body suits during clinic visits to capture entire […]
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 muscle cells.  Since the most commonly used viruses for these purposes are AAVs (Adeno-Associated Viruses), many presentations covered AAVs at the 24th International Annual Congress […]
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 included a talk outlining the different ways scientists are trying to understand how the brain can be affected in cases of Duchenne muscular dystrophy (DMD). […]
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 registered participants, 4 days of research talks, and over 570 research posters spread out among 5 different buildings.  As usual, updates in Duchenne Muscular Dystrophy […]
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 research conference focused solely on neuromuscular diseases.  We are here because it’s an opportunity to hear about the latest research developments and network with others […]
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 to helping those with Duchenne and are confident that Doug Ingram and his team will not let this slow them down. In drug development there […]
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 of Bamboo Therapeutics, a Cure Duchenne funded company. Pfizer’s preliminary data is very encouraging despite two SAE’s which resolved with approved treatment. We are eager […]
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 Pfizer’s Duchenne gene therapy trial, to founding the first Duchenne gene editing company, Exonics (recently acquired by Vertex), to investing in crucial AAV manufacturing in […]
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 the haystack. Only sixteen years later and big pharma and biotech are now competing to be leaders in the Duchenne space. Clinical trials for Duchenne […]
WAVE Open Letter to Community
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