PTC Therapeutic, Roche Announce Joint Program in Spinal Muscular Atrophy Enters First Stage of Clinical Development

PTC Therapeutics, Inc. PTCT, the SMA Foundation, and Roche RHHBY, announced today that their joint research program in Spinal Muscular Atrophy (SMA) has entered the first stage of clinical development aiming to assess safety and tolerability of an oral compound in healthy volunteers. SMA is a genetic neuromuscular disorder that leads to muscle weakness and mobility impairment and is considered the leading genetic cause of death in infants and toddlers.    See full press release