Spinal Muscular Atrophy clinical trials at UC Irvine
2 research studies open to eligible people
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
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 Namita Goyal, MD.