Spinal muscular atrophy

rare congenital neuromuscular disorder

Nusinersen, marketed as Spinraza, is a medication used in treating spinal muscular atrophy (SMA), a rare neuromuscular disorder. In December 2016, it became the first approved drug used in treating this disorder.

Biogen Inc. is an American multinational biotechnology company based in Cambridge, Massachusetts, United States specializing in the treatment of neurological diseases. The company's primary products are dimethyl fumarate (Tecfidera), diroximel fumarate (Vumerity), interferon beta-1a (AVONEX), peginterferon beta-1a (Plegridy), and natalizumab (Tysabri), all for the treatment of multiple sclerosis (collectively 45% of 2024 revenues); nusinersen (Spinraza) for the treatment of spinal muscular atrophy (16.2% of 2024 revenues); omaveloxolone (Skyclarys) for the treatment of Friedreich's ataxia (4.0

gene therapy product

Risdiplam, sold under the brand name Evrysdi, is a medication used to treat spinal muscular atrophy (SMA) and is the first oral medication approved to treat this disease by the US Food and Drug Administration (FDA).

German neurologist (1857–1919)

Survival of motor neuron 1 (SMN1), also known as component of gems 1 or GEMIN1, is a gene that encodes the SMN protein in humans.The chemical formula is:C1394H2154N384O438S13

Survival of motor neuron 2 (SMN2) is a gene that encodes the SMN protein (full and truncated) in humans.

Austrian physician

group of diseases

InterPro Domain