PTC Therapeutics Announces Positive Preliminary Clinical Data from the FIREFISH Study in Type 1 SMA Patients

The clinical data were presented at the annual meeting of the American Academy of Neurology. The presentation from Part 1, the Dose Finding section, of the ongoing FIREFISH study. The open-label study investigated RG7916, an oral survival motoneuron 2 (SMN2) splice modifier, in type 1 SMA patients. The data from… Att fortsätt läsa