PTC Therapeutics (NASDAQ:PTCT) has been coming along well as ... whereby patients receiving votoplam achieved reduction of blood HTT protein in a dose-dependent manner. Beyond this, the 12-month data ...

A first-in-class, brain-penetrant sGC stimulator, zagociguat is hypothesized to rebalance dysregulated cellular pathways in MELAS. By restoring cellular functions that support mitochondria, zagociguat ...