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Drug Primer

Evrysdi, a survival of motor neuron 2 (SMN2) gene splicing modifier, was approved by the US Food and Drug Administration for the treatment of spinal muscular atrophy in patients 2 months of age and older.

Ofatumumab (Kesimpta®; Novartis Pharmaceuticals Corporation), C20-directed cytolytic antibody, has recently been approved for the treatment of adult patients with relapsing forms of multiple sclerosis, including clinically isolated syndrome, relapsing-remitting disease, and active secondary progressive disease.

In August 2020, the US Food and Drug Administration (FDA) approved a new therapy for the treatment of Duchenne muscular dystrophy (DMD) in patients who have a confirmed mutation in the dystrophin gene that is amenable to exon 53 skipping. Viltolarsen, marketed under the brand name Viltepso by NS Pharma, is an antisense oligonucleotide of…