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Spinal Muscular Atrophy Program Advances with Additional Product Candidate Entering Clinical Development

Spinal Muscular Atrophy Program Advances with Additional Product Candidate Entering Clinical Development (NASDAQ:PTCT)

SOUTH PLAINFIELD, N.J., Jan. 7, 2016 /PRNewswire/ — PTC Therapeutics, Inc. (Nasdaq: PTCT) today announced that RG7916, an additional SMN2 splicing modifier from the company’s joint development program with Roche and the SMA Foundation in spinal muscular atrophy (SMA), has entered clinical development.  RG7916 is designed to shift SMN2 pre-mRNA splicing toward the production of full length SMN mRNA.  A Phase 1 study in healthy volunteers has been initiated to investigate the safety, tolerability, pharmacokinetics and pharmacodynamics of RG7916.

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