Long-term Follow-up Study of Risdiplam in Participants With Spinal Muscular Atrophy (SMA)

• Clinical diagnosis of SMA
• Prescribed or continued risdiplam based on clinical judgment of prescriber, as per the Evrysdi® USPI, after U.S. FDA approval (07 August 2020)

Sub study:

Participants in the main study (ML43702) are eligible to be included in the sub study only if all of the following criteria apply:

• Age ≥ 10 years at the time of signing Informed Consent Form
• Willingness and ability to use smartphone technology
• Fluency in English (written and spoken as per the judgment of the investigator)
• Willingness and ability to complete all aspects of the sub study, including respiration and swallowing measurements using respiratory inductance plethysmography (RIP) belts and surface electromyography (sEMGs)
• Hammersmith Functional Motor Scale-Expanded (HFMSE) > 10
• Functional Oral Intake Scale (FOIS) >1
• Willingness to be video recorded during in-clinic SMA-DAT tasks and ADAM sensor assessments and training
• Availability of a caregiver who is willing to participate throughout this sub study

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

Sub study:

Potential participants will be excluded from the sub study if they meet any of the following criteria:

• Current respiratory infection that, in the opinion of the investigator, would interfere with the conduct of the sub study
• History or known presence of any significant psychiatric disorder such as schizophrenia, bipolar disorder, or substance use disorders
• Current active clinically significant anxiety or depressive disorder, as judged by the investigator, that is likely to impede a participant's ability to participate in the sub study
• Wearing a pacemaker (due to incompatibility with the ADAM sensor)
• Inability to tolerate the performance of sub study procedures