Real-World Motor Improvements in SMA from Nusinersen

The greatest motor gains were observed in the first 14 months for patients younger than 2 years, although many still require respiratory support and feeding assistance.

Previously unattainable gains in spinal muscular atrophy (SMA) have been made since the approval of disease-modifying therapies, particularly if started presymptomatically. These researchers used European registry data to investigate the durability of treatment benefit from nusinersen for symptomatic patients with SMA. For up to 38 months, they followed 143 infants and children who could not sit independently at the start of treatment, divided by age: ≤2 years old at treatment initiation (72.7% had 2 SMN2 copies; mean follow-up, 23 months) and >2 years old (69.0% had 2 or 3 SMN2 copies; mean follow-up, 28 months). The researchers used the Children's Hospital of Philadelphia Infant Test of Neuromuscular Disorders (CHOP INTEND), which assesse…

Reviewing Author

Leana Doherty, MD

Disclosures

Editorial BoardsNeurohospitalist Society; InterACTN (Annals of Clinical and Translational Neurology)

Leana Doherty, MD

Disclosures

Editorial BoardsNeurohospitalist Society; InterACTN (Annals of Clinical and Translational Neurology)

Real-World Motor Improvements in SMA from Nusinersen

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Reviewing Author