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Risdiplam-Treated Infants with Type 1 Spinal Muscular Atrophy versus Historical Controls

  • Darras B
  • Masson R
  • Mazurkiewicz-Bełdzińska M
  • et al.
New England Journal of Medicine (2021) 385(5) 427-435
DOI: 10.1056/nejmoa2102047
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Abstract

Abstract Background Type 1 spinal muscular atrophy (SMA) is a progressive neuromuscular disease characterized by an onset at 6 months of age or younger, an inability to sit without support, and def...

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APA

Darras, B. T., Masson, R., Mazurkiewicz-Bełdzińska, M., Rose, K., Xiong, H., Zanoteli, E., … Servais, L. (2021). Risdiplam-Treated Infants with Type 1 Spinal Muscular Atrophy versus Historical Controls. New England Journal of Medicine, 385(5), 427–435. https://doi.org/10.1056/nejmoa2102047

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