Long-Term Follow-Up Study of Risdiplam in Participants With Spinal Muscular Atrophy (SMA)

Neurology
Jeffrey Mullen
Long-Term Follow-Up Study of Patients With Spinal Muscular Atrophy Receiving Risdiplam Treatment
Brain - Neurologic/ Psychologic
Muscles and Bones - Musculoskeletal
Spinal Muscular Atrophy

Study Description

Eligibility

  1. Confirmed diagnosis of 5q-autosomal recessive SMA
  2. Prescribed or continued risdiplam based on clinical judgment of prescriber, as per the Evrysdi® USPI, after U.S. FDA approval (07 August 2020)
  1. Hypersensitivity to risdiplam
  2. Participated in a registrational trial for risdiplam (i.e., Firefish [NCT02913482], Sunfish [NCT02908685], Jewelfish [NCT03032172], and Rainbowfish [NCT03779334])
  3. Aged greater than or equal to 2 years with a score less than or equal to 3 on the HFMSE or RULM at study entry
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