dm+d

Unassigned

New Medicines

Spinal muscular atrophy

Information

New molecular entity
Scholar Rock
Scholar Rock

Development and Regulatory status

None
Phase III Clinical Trials
Phase III Clinical Trials
Yes
Yes

Category

A fully human anti-proMyostatin monoclonal antibody (mAb) of the immunoglobulin G4 (IgG4)/lambda isotype that binds to human pro/latent myostatin with high affinity, given every 4 weeks.
The estimated incidence is between 1 in 6,000 and 1 in 10,000 live births and the carrier frequency is between 1 in 40 and 1 in 60 [1].
Spinal muscular atrophy
Intravenous infusion