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  • AveXis Submits BLA for SMA Gene Therapy AVXS-101 to FDA October 18, 2018
    This week, AveXis, a Novartis company, reported that it has submitted a Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA) requesting approval of AVXS-101 to treat infants with spinal muscular atrophy (SMA) type 1. AveXis is also submitting regulatory applications for the therapy in Europe and Japan. If approved, AVXS-101 would […]
  • Pharnext Announces Positive Results from Phase 3 Trial Using PXT3003 to Treat CMT1A October 16, 2018
    Today, French pharmaceutical company Pharnext SA announced positive topline results from its pivotal Phase 3 clinical trial (PLEO-CMT) evaluating two doses of PXT3003 compared to placebo during 15 months for the treatment of Charcot-Marie-Tooth disease type 1A (CMT1A). PLEO-CMT was a pivotal, 15-month, double-blind Phase 3 study that assessed the efficacy and safety of PXT3003 compared […]
  • For MDA Executive Director Patrick Cusick, a Diagnosis Led to a New Direction October 12, 2018
    Patrick Cusick’s journey with MDA — and with muscular dystrophy — started 12 years ago, in, of all places, a life insurance office. Twenty-nine at the time, the Ohio native and his wife were expecting their first child, and making preparations as parents (or soon-to-be parents) do. Securing life insurance meant taking some medical tests; […]
  • Sarepta Therapeutics Announces Positive Updated Results from Gene Therapy Trial for Duchenne Muscular Dystrophy October 11, 2018
    This week, at the 23rd International Congress of the World Muscle Society in Argentina, Dr. Jerry Mendell of Nationwide Children’s Hospital in Columbus, Ohio, shared additional data relating to Sarepta Therapeutics’ AAVrh74.MHCK7.micro-Dystrophin gene therapy program for Duchenne muscular dystrophy (DMD).  Dr. Mendell had previously presented results for the first three patients in the study back in […]
  • Researchers at SUNY Binghamton Seek Participants for BMD Biomarker and Patient-Reported Outcomes Study October 4, 2018
    Researchers at the State University of New York at Binghamton’s School of Pharmacy and Pharmaceutical Sciences are seeking participants living with Becker muscular dystrophy (BMD) to participate in a biomarker and patient-reported outcomes study. The purpose of the Becker Muscular Dystrophy Biomarker and Patient-Reported Outcomes Study is to learn more about disease progression in patients […]

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