Sarepta has completed the ESSENCE study, which is a confirmatory trial aimed at supporting full FDA approval of the exon-skipping PMOs Amondys 45 and Vyondys 53. The study did reinforce […]
CureDuchenne is sharing a community letter from Sarepta announcing a strategic company restructuring and pipeline prioritization plan. Additionally, Sarepta shared that the U.S. FDA has requested and Sarepta has agreed to include a […]
Dr. Gilmore O’Neill, Chief Medical Officer of Sarepta Therapeutics, Inc, gave a scientific overview of Sarepta’s RNA-targeted exon skipping technologies, PMO and PPMO. Dr. O’Neill provided valuable information on this […]
The second day of the conference focused on gene editing, gene therapy as well as the latest research into the development of disease biomarkers. Presentations included: Dongsheng Duan (University of […]
CureDuchenne staff is participating in the New Direction in Biology and Disease of Skeletal Muscle Conference in New Orleans this week. Our staff scientist, Michael Kelly, will be providing a summary […]
After waiting for a very long time, the Duchenne community now has clarity on the Advisory Committee meetings dates for BioMarin and Sarepta. BioMarin’s date is November 24, 2015 […]
We couldn’t be prouder that CureDuchenne has played a critical role in the development of what we hope will be the first-ever pharmaceutical treatments for Duchenne muscular dystrophy. Investments by […]
CureDuchenne is proud to have supported all types of Duchenne research over the past 10 years. That is why CureDuchenne has been a leader in funding exon skipping, stop codon, […]
There continues to be good news for the Duchenne community. We will continue to monitor Duchenne research news and share it with you as it becomes available. Here are three […]
