2 results
Approved WMORecruiting
To evaluate the efficacy of PTC857 in reducing disease progression in subjects with amyotrophic lateral sclerosis (ALS)
Approved WMORecruiting
The objective of this open-label extension (OLE) study is to assess the effect of long-term safety and treatment effect of Voxelotor in participants who have completed treatment in Study GBT440-031, using the following parameters:- Safety based upon…