Rare Disease Research, LLC | Atlanta, GA
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About
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. Participants who discontinue risdiplam may still remain in the study, if they agree to continue participating in the follow-up assessments.
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Primary purpose
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Interventional model
Masking
500 participants in 1 patient group
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Central trial contact
Reference Study ID Number: ML43702 https://forpatients.roche.com/
Data sourced from clinicaltrials.gov
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