New risdiplam data show improvements in developmental milestones

PTC Therapeutics Inc. (NASDAQ:PTCT) and Genentech Inc. reported updated data from part I of the two-part Phase II/III FIREFISH and SUNFISH trials of risdiplam (RG7916) to treat spinal muscular atrophy (SMA) that highlighted improvements in developmental milestones and motor function.

Risdiplam's oral delivery could offer an advantage over marketed SMA drug Spinraza nusinersen from Biogen Inc. (NASDAQ:BIIB), which is a survival of motor neuron 2 centromeric (SMN2) splicing modifier delivered by injection into the spine. Risdiplam is an oral SMN2 splicing modifier...