Guest blog by Tiffany L. Cook, MS, CCC-SLP Words of advice from Wil’s elementary school Occupational Therapist, Jessica Berger, MOT, OTR/L, “When there are little breaks taken throughout the day, […]
Data show for the first time a systemic therapeutic effect in DMD dogs using a rAAV2/8 micro-dystrophin gene therapy approach without immunosuppressive treatment CAMBRIDGE, Mass., July 27, 2017 (GLOBE NEWSWIRE) […]
CureDuchenne is proud to announce and welcome Tiffany Cook as its new Family Resource Manager. She, along with the CureDuchenne team, will be enhancing and presenting family education materials and […]
Fourteen years ago, I travelled to Russia with my family, just months after our son Hawken had been diagnosed with Duchenne muscular dystrophy. It was very raw for us at […]
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 […]
Duchenne organizations are invited to fund CRISPR/Cas9 research at Exonics Therapeutics in a venture philanthropy collaboration. We are making progress on treatments for Duchenne, but a cure is the goal […]
This webinar provided an update from Santhera Pharmaceuticals about the SIDEROS trial of idebenone in boys taking steroids. Santhera also provided an update about their regulatory efforts to bring this treatment to boys with Duchenne in the US and Europe.
Liestal, Switzerland, June 22, 2017 – Santhera Pharmaceuticals (SIX: SANN) announces that the UK`s Medicines and Healthcare products Regulatory Agency (MHRA) has granted Raxone (idebenone) a positive scientific opinion through the Early […]
CAMBRIDGE, Mass. and EVRY, France, June 21, 2017 (GLOBE NEWSWIRE) — Sarepta Therapeutics, Inc. (NASDAQ:SRPT), a U.S. commercial-stage biopharmaceutical company focused on the discovery and development of unique RNA-targeted therapeutics […]
