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 […]
Each year on Father’s Day, I like to take a moment and reflect on the memories I have with my family, specifically with my son Hawken. Being a father to […]
Father’s Day is the perfect time to reflect on all that our fathers do for us. For boys with Duchenne, it’s a day that they get to cherish their fathers […]
NEWPORT BEACH, Calif., June 15, 2017 – RASRx announced today that the United States Food and Drug Administration (FDA) has granted an Orphan Drug Designation (ODD) for its compound RASRx1902 […]
NEWPORT BEACH, Calif., June 14, 2017 – CureDuchenne, a nonprofit dedicated to funding research and improving patient care for Duchenne muscular dystrophy, will host a webinar with Santhera Pharmaceuticals on […]
Academia has long been an incubating source of incredible medical breakthroughs. Sometimes it takes a little push and risk-taking to take that progressive research out of the lab and into […]
