June 1, 2021 VISION-DMD 24-week study readout: Update from Santhera Dear U.S. Duchenne community, We are happy to provide an update of the VISION-DMD Phase 2b study conducted by Santhera […]
In 2013, our oldest son, Joshua, was diagnosed with Duchenne muscular dystrophy. He was 5 years old at the time and we were devastated with the diagnosis. After doing some […]
Our son was diagnosed April 1, 2019. We have spent the past 2 years doing research, completing a clinical trial and being an advocate for our son and Duchenne Muscular […]
We are the Edwards Family in North Dakota. Our 11 year old son Grant has Duchenne. Diagnosed at age 4. Grant’s younger brother Christian age 8 is unaffected. I carry […]
We have two sons and a daughter and no family history of Duchenne. In April of 2018, we took our kids to the pediatrician for a well check. We mentioned […]
Results from the first 11 participants enrolled in Study 9001-103 ENDEAVOR showed robust transduction, delivering mean vector genome copies of 3.87 per nucleus Treated patients achieved mean micro-dystrophin expression levels […]
With recent clinical updates from Pfizer, Sarepta and Audenteson their programs in muscular disorders, the timely Gene Therapy for Muscular Disorders arrives as the only meeting dedicated to overcoming the translational […]
Update on Pfizer’s Duchenne gene therapy Phase 3 clinical program In line with our continued efforts to work with the Duchenne community and share information as it becomes available, we […]
Results suggest a highly potent next-generation treatment that could offer greater efficacy with less frequent dosing SRP-5051 dosed monthly at 30 mg/kg delivered mean exon skipping of 10.79% and mean dystrophin […]
