MYOSTATIN INHIBITION PLATFORM
Myostatin is a natural protein that normally works to regulate skeletal muscle growth, an important process in healthy muscular development. In patients with neuromuscular diseases, over-active myostatin can critically limit the growth needed to achieve normal developmental and functional milestones. Myostatin inhibition is a potential therapeutic strategy for children and adults with a range of neuromuscular conditions for whom active myostatin can limit the skeletal muscle growth needed to achieve developmental and functional milestones.
Spinal Muscular Atrophy (SMA)
A Study to Evaluate the Efficacy and Safety of Taldefgrobep Alfa in Participants With Spinal Muscular Atrophy (RESILIENT)
Drug: Taldegrobep Alfa | Age: 4-21 | Phase: 3
