Isembyld, containing the active substance apitegromab‑mstn, is the first approved treatment for SMA that targets muscle loss. The approval covers treatment of patients from two years of age who are already receiving other therapies for the disease.
SMA affects approximately 1 in 10,000 live births and is one of the leading genetic causes of infant mortality. The disease is caused by a defective SMN1 gene that cannot produce the protein necessary for motor neuron survival, leading to progressive muscle weakness and wasting.
BREAKING