About the Investigational Medication
What is Taldefgrobep alfa?
What is Taldefgrobep alfa?
Taldefgrobep alfa is an anti-myostatin, which means it targets myostatin, a natural protein that is important in healthy muscular development. In people with neuromuscular diseases, active myostatin can limit the skeletal muscle growth needed to achieve developmental and functional milestones.
In preclinical models, the inhibition of myostatin increased muscle mass and improved muscle function. Taldefgropep alfa is designed to increase muscle mass and strength in SMA patients via dual inhibition functioning, by directly lowering myostatin levels and blocking myostatin function.
Data from prior clinical studies in healthy volunteers and Duchenne Muscular Dystrophy suggest that taldefgrobep alfa is generally safe and well-tolerated.
Administration of the Study Medication
Taldefgrobep alfa, or placebo, will be administered via injection under the skin once weekly during clinic visits or at the home.
After the Baseline visit, participants will attend visits at the site clinic approximately every 12 weeks preferably in the morning.
