Spinal Muscular Atrophy (SMA)

Also known as: Spinal Muscular Atrophy / Spinal muscular atrophy NOS / Muscular Atrophy, Spinal / Spinal muscular atrophy, unspecified / Spinal amyotrophy

DrugDrug NameDrug Description
DB13161NusinersenA medication used to treat spinal muscular atrophy.
DB15528Onasemnogene abeparvovecA gene therapy used to treat neonatal and infant patients with spinal muscular atrophy caused by bi-allelic mutations in the survival motor neuron 1 (SMN1) gene.
DB15305RisdiplamAn oral mRNA splicing modifier used in the treatment of spinal muscular atrophy (SMA).