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Lysogene and Sarepta Therapeutics Announce Dosing of the First Patient in AAVance, a Phase 2/3 Clinical Trial Investigating LYS-SAF302, a Gene Therapy for the Treatment of MPS IIIA (Sanfil...

Sarepta Therapeutics, Inc. (SRPT) 
Last sarepta therapeutics, inc. earnings: 2/26 04:05 pm Check Earnings Report
Company Research Source: GlobeNewswire
-- Trial to assess efficacy on neurodevelopmental status of MPS IIIA patients -- CAMBRIDGE, Mass. and PARIS, Feb. 14, 2019 (GLOBE NEWSWIRE) -- Lysogene (FR0013233475 – LYS), a pioneering biopharmaceutical company specializing in gene therapy targeting central nervous system (CNS) diseases, and Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, announced today that the first patient has been dosed in AAVance, a global Phase 2-3 clinical trial of LYS-SAF302, a gene therapy for the treatment of Mucopolysaccharidosis Type IIIA (MPS IIIA). AAVance is a single-arm trial aimed at evaluating the effectiveness of a one-time delivery of a recombinant adeno-associated virus vector rh.10 carrying the N-sulfoglucosamine sulfohydrolase (SGSH) gene. MPS IIIA is caused by mutations in the SGSH gene, which is involved in producing an enzyme necessary for the breakdown and disposal of long chain sugar molecules. LAF-SAF302 is intended to deliver a Show less Read more
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