Risdiplam for very young children after gene therapy for SMA
Treatments studied
Part of Brain & nervous system clinical trials.
This trial studies whether risdiplam, given early after gene therapy, is safe and helps children with spinal muscular atrophy (SMA) keep improving or stay stable. It focuses on children under 2 years old who already received gene therapy and need to meet timing and health requirements.
Summary written for real people, not researchers, by Clin2.
Who can take part
- Be under 2 years old when you sign consent
- Have confirmed 5q autosomal recessive SMA based on genetic testing
- Have exactly two working copies of the SMN2 gene (confirmed by lab tests)
- Have already received onasemnogene abeparvovec gene therapy between about 13 and 30 weeks before starting this study
- Not have had major lung/fever illness right around screening (such as pulmonary hospitalization in last 2 months or fever in last week)
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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Other trials that look related to this one.
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.
This trial tests if the drug salanersen can help movement and is safe when given to babies diagnosed with spinal muscular atrophy before they show symptoms. It's for very young infants with specific genetic types of SMA.
This study looks at how muscles work in children and young adults with spinal muscular atrophy (SMA) who are taking or starting SMN-directed therapies (like Evrysdi, Spinraza, or Zolgensma). It aims to understand the body's response to these treatments, which could help improve future care.
This study follows children with spinal muscular atrophy (SMA)—a genetic condition causing muscle weakness—who are being treated with a medicine called risdiplam. Researchers want to understand how well the treatment works and how it affects daily life in young patients.
This study tests a new medicine called apitegromab for babies under 2 years old with spinal muscular atrophy (SMA). It looks at whether the drug can help with muscle strength and reaching motor milestones like sitting or rolling.
This trial tests a one-time gene therapy given through a vein to add a working SMN gene in children with spinal muscular atrophy (SMA). It mainly looks at safety first, and also checks early signs that treatment helps the disease.
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