Kevin Flanigan, MD is a leading expert in the development of gene therapies for Duchenne, and the director of the Center for Gene Therapy in the Abigail Wexner Research Institute […]
Entrada Therapeutics announced encouraging new data on their exon 44 skipping program following their presentation at TIDES USA 2022. CureDuchenne provided funding to Entrada last year as part of their Series […]
National Center of Neurology and Psychiatry and Nippon Shinyaku announce positive results for a Phase I/II study evaluating the efficacy and safety of their exon 44 skipping drug (NS-089/NCNP-02) Results […]
**UPDATE** Duchenne gene therapy developers and scientific experts discuss AAV in DMD: Antitransgene SAEs. Please watch this webinar to learn more about recent concerns in gene therapy clinical trials. **ORIGINAL POST** […]
CureDuchenne continued funding a robust Duchenne drug development pipeline with a new investment in Entrada Therapeutics as part of their latest financing from a group of investors. Entrada Therapeutics’ promising […]
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 […]
CureDuchenne hosted a webinar with Dr. Paula Clemens and Lauren Morgenroth on the NS Pharma Exon 53 Skipping Program. The webinar was an opportunity for the Duchenne community to learn […]
Dr. Kevin Flanigan from Nationwide Children’s Hospital provided an update on his Dup2 research during a CureDuchenne hosted call with families on September 6. Back in 2010, Dr. Flanigan told […]
If you live in Texas, are amendable to exon skipping 51, and you are having difficulty accessing Exondys 51, you or your provider may file a complaint to HPM_Complaints@hhsc.state.tx.us. Exondys […]
