The effect of this medication on strength, walking ability and activities of daily living is being evaluated for this fairly rare progressive adult neuromuscular disorder. A label aspect has begun (beginning 6/2013) as patients complete the initial study they are invited to continue in this new phase.
Timescale: 06/01/2012 - 08/30/2021 Current status: Project live
Primary Investigator: Eileen Fowler
