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


About this study

A multi-center, longitudinal, prospective, non-comparative study to investigate the long-term safety and effectiveness of risdiplam, prescribed based on clinician judgment as per the Evrysdi® U.S. Package Insert (USPI) in adult and pediatric participants with spinal muscular atrophy (SMA). In this study, participants will be followed for up to 5 years from enrollment or until withdrawal of consent, loss to follow-up, or death.

Participation eligibility

Participant eligibility includes age, gender, type and stage of disease, and previous treatments or health concerns. Guidelines differ from study to study, and identify who can or cannot participate. There is no guarantee that every individual who qualifies and wants to participate in a trial will be enrolled. Contact the study team to discuss study eligibility and potential participation.

Inclusion Criteria:

- Confirmed diagnosis of 5q-autosomal recessive SMA

- 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:

- Hypersensitivity to risdiplam

- Participated in a registrational trial for risdiplam (i.e., Firefish [NCT02913482],
Sunfish [NCT02908685], Jewelfish [NCT03032172], and Rainbowfish [NCT03779334])

Note: Other protocol defined Inclusion/Exclusion criteria may apply.

Eligibility last updated 12/7/22. Questions regarding updates should be directed to the study team contact.

Participating Mayo Clinic locations

Study statuses change often. Please contact the study team for the most up-to-date information regarding possible participation.

Mayo Clinic Location Status Contact

Rochester, Minn.

Mayo Clinic principal investigator

Duygu Selcen, M.D.

Open for enrollment

Contact information:

Iesha Abbajebel

(507) 293-0545


More information


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