PTC Therapeutics has released preliminary data from its ongoing clinical studies, FIREFISH and SUNFISH. These studies are investigating whether RG7916 (risdiplam) could be a potential treatment for spinal muscular atrophy (SMA).
So far, the FIREFISH study has shown that infants with Type 1 SMA treated with RG7916 have reached new developmental milestones (e.g head control and sitting). The SUNFISH study – which is focussed on people with Type 2/3 SMA – has also shown that RG7916 can improve motor function.
No severe adverse events have been reported to date, suggesting that the drug is safe and well-tolerated.
For further information please read PTC Therapeutics’ press release.