Archives for April 2014

Thank you for believing in us and SOPHIA!!!

We are very proud to announce enrollment for the Gene Therapy Clinical Trial is now OPEN. Phase I Clinical Trial of Systemic SMN Gene Therapy For Spinal Muscular Atrophy To Begin at Nationwide Children’s Hospital in May. The purpose of this trial is to evaluate safety and efficacy of intravenous delivery of self-complementary scAAV9.CB.SMN as a treatment of Spinal Muscular Atrophy type 1 (SMN1). This was a program Sophia's Cure Foundation lead the way in with a multi-million dollar … [Read more...]

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