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 […]
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 […]
