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
With a flurry of recent activity, 17 trial sites are now open for enrollment. We are especially excited to share that our first international sites in Canada and Australia have launched. Sites are also expected to launch soon in Europe and Israel.
SRPT press release – golodirsen NDA acceptance 02 14 19
We are excited about the UT Southwestern Wellstone Symposium on March 30! Don’t forget to RSVP by March 1. We’ll see you there!
Thursday February 28th at 4pm Dr. Eric Olson is giving a talk at the President’s Lecture at UT Southwestern (South Campus Gooch Auditorium) and will be highlighting the DMD project. Steve Gray, PhD is also speaking about gene therapy. The lecture is open to the general public.
We are excited that our industry partner, NS Pharma, has applied for accelertated approval for it’s exon 53 skipping therapy, viltolarsen. We applaud the dedication to the Duchenne community by NS Pharma and look forward to continued success. NS Pharma Community Letter