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RG7916 Granted Orphan Drug Designation in the U.S. for the Treatment of Spinal Muscular Atrophy

SOUTH PLAINFIELD, N.J., Jan. 6, 2017 /PRNewswire/ — PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation (ODD) to RG7916 for the treatment of patients with Spinal Muscular Atrophy (SMA). RG7916 is part of PTC’s joint development program in SMA with Roche and the SMA Foundation (SMAF). SMA is a rare genetic disorder that results in neuromuscular disability beginning in infancy and is the leading inherited cause of mortality in infants and young children.

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