PTC Therapeutics: Solid data from its oral product Risdiplam in spinal muscular atrophy

PTC Therapeutics’ (PTCT) product Risdiplam demonstrated safety and tolerability at all the doses used in the studies on Type 1, 2 and 3 spinal muscular atrophy (SMA).

The good news emanated from interim data of the Part 1, open-label studies of FIREFISH and SUNFISH trials, with the oral drug Risdiplam on SMA patients. Risdiplam was dubbed effective,  substantiated by a confirmed increase in motor function.

Stuart W. Peltz, Ph.D., Chief Executive Officer of PTC Therapeutics explained that the emerging results . . .

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