Spinal Muscular Atrophy clinical trials at UC Irvine

2 research studies open to eligible people

Showing trials for

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

    open to all eligible people

    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.

    Orange, California and other locations

  • Clinical Procedures to Support Research in ALS

    open to eligible people ages 18 years and up

    The purpose of the Clinical Procedures To Support Research (CAPTURE) study is to utilize information collected in the medical record to learn more about a disease called amyotrophic lateral sclerosis (ALS) and related disorders.

    Irvine, California and other locations

Our lead scientists for Spinal Muscular Atrophy research studies include .

Last updated: