PTC's oral SMA candidate improves motor development scores in pivotal trial

PTC Therapeutics Inc. (NASDAQ:PTCT) reported updated interim data from part 1 of the pivotal Phase II/III FIREFISH trial showing that oral risdiplam (RG7916) improved motor development scores in infants with spinal muscular atrophy (SMA) type 1.

In 11 evaluable patients in part 1, risdiplam increased Children's Hospital of Philadelphia Infant Test of Neuromuscular Disorders (CHOP-INTEND) scores -- a measure of motor milestone development -- by a median of 14 points from baseline to day 182. Of those patients, 91% had a greater than four-point increase in CHOP-INTEND score from baseline to day 182...