Risdiplam for young children with SMA after gene therapy plateau
Treatments studied
Part of Brain & nervous system clinical trials.
This trial studies whether risdiplam is safe and helpful for children under 2 with spinal muscular atrophy (SMA) whose function has leveled off or declined after gene therapy. It may be an option if you saw benefits from gene therapy but then noticed a stop or setback in key abilities.
Summary written for real people, not researchers, by Clin2.
Who can take part
- Your child is under 2 years old when starting the screening/consent
- Your child has genetically confirmed 5q-related SMA (SMN1 gene problem) and is confirmed to have 2 copies of the SMN2 gene
- Your child already received onasemnogene abeparvovec (gene therapy) at least 13 weeks ago
- After gene therapy, your child shows a plateau or decline within the last 26 weeks, including trouble with swallowing plus at least one other function issue
- Your child has not recently joined other experimental drug studies and has no major uncontrolled lab problems from the gene therapy guidance
- Your child does not need a breathing machine or a tracheostomy, and does not have a feeding tube with an OrSAT score of 0
Quick eligibility check
Answer a few plain-language questions, based on this study's own requirements, to get a preliminary sense of fit.
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