The Medical Letter on Drugs and Therapeutics
Nusinersen (Spinraza) for Spinal Muscular Atrophy
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The FDA has approved nusinersen (Spinraza – Biogen) for treatment of spinal muscular atrophy (SMA), a hereditary neurodegenerative disease that occurs in about one in every 10,000 births.1 It is the first drug to be approved in the US for this indication.

SPINAL MUSCULAR ATROPHY — SMA is an autosomal recessive neuromuscular disorder in which degeneration of alpha motor neurons in the anterior horn cells of the spinal cord leads to muscle weakness and atrophy. Most cases are caused by a homozygous deletion or mutation in the 5q13 survival of motor neuron 1 (SMN1) gene, which leads to SMN protein deficiency. Mutations in a similar gene, SMN2, are correlated with a milder SMA phenotype.2

SMA is classified based on the age of symptom onset and motor function achieved. Infants with type 1 SMA (age of onset 0-6 months) are unable ... more      

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Title: Nusinersen (Spinraza) for Spinal Muscular Atrophy
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