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

Official Title

Long-Term Follow-Up Study of Patients With Spinal Muscular Atrophy Receiving Risdiplam Treatment

You can join if...

Inclusion Criteria:

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)

Exclusion Criteria:

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