Long-term Follow-up Study of Patients With Spinal Muscular Atrophy Receiving Risdiplam Treatment
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 SMA. In this study, participants will be followed for the duration of the study or until withdrawal of consent, loss to follow-up, or death. Participants who discontinue risdiplam may still remain in the study, if they agree to continue participating in the follow-up assessments. An optional sub study will assess the feasibility, acceptability, and adherence of remote assessment of motor and bulbar functions in participants with SMA using wearable and smartphone-based biosensors. Approximately 39 participants from the main study are planned to be enrolled in the sub study.
• 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