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February 24, 2021New England Journal of MedicineOpen Access

Risdiplam in Type 1 Spinal Muscular Atrophy

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Authors

GBGiovanni BaranelloGreat Ormond Street HospitalBDBasil T. DarrasUniversità Cattolica del Sacro CuoreJDJohn DayGeneral Cardiology

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Baranello et al. (2021) studied this question.

synapsesocial.com/papers/69d83ec2f4e559c61eae31achttps://doi.org/10.1056/nejmoa2009965
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Also Consider

Synapse has enriched 5 closely related papers on similar clinical questions. Consider them for comparative context:

  1. 1Risdiplam distributes and increases SMN protein in both the central nervous system and peripheral organs2018 · 175 citations
  2. 2Special Considerations in the Respiratory Management of Spinal Muscular Atrophy2009 · 163 citations
  3. 3A phase 1 healthy male volunteer single escalating dose study of the pharmacokinetics and pharmacodynamics of risdiplam (RG7916, RO7034067), a SMN2 splicing modifier2018 · 109 citations
  4. 4Developmental milestones in type I spinal muscular atrophy2016 · 140 citations
  5. 5Natural history of infantile‐onset spinal muscular atrophy2017 · 421 citations