Gene therapy for spinal muscular atrophy in low-resource settings
Part of Brain & nervous system clinical trials.
This trial tests a new gene therapy called Vesemnogene Lantuparvovec for people with spinal muscular atrophy (SMA). It is designed for patients in low- and middle-income countries who cannot access or did not respond to other treatments. The goal is to see if the therapy is safe and effective in real-world conditions.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You must have a confirmed genetic diagnosis of SMA (a specific type of gene change).
- Your doctor must believe the study treatment could help you based on your SMA type and condition.
- You cannot have had other curative SMA treatments that worked, or you cannot access them.
- Your liver function must be within a normal range (specific blood tests required).
- You must have a low level of antibodies against the virus used in the gene therapy (measured by a blood test).
- You must be able to have a spinal injection (intrathecal therapy).
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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This trial tests a new medicine called EXG001-307 for babies with spinal muscular atrophy (SMA) types 1 and 2. The goal is to see if it is safe and helps with muscle strength and breathing.
This early-stage study tests whether a one-time gene therapy can safely improve outcomes for babies with the most severe type of spinal muscular atrophy (SMA type 1). It focuses on safety and early signs of benefit, and participation requires specific genetic and health criteria.
This study tracks how well the gene therapy ITVISMA works in everyday medical practice for people with spinal muscular atrophy (SMA). It aims to see if the treatment is safe and effective in a real-world setting.
This trial tests a new gene therapy called GB221 for infants with SMA type 1, a rare muscle-weakening disease. It aims to see if this treatment can improve muscle strength and development, and it is for babies who have not had prior gene therapy.
This trial tests a new gene therapy called GC101 given as a one-time injection into the spinal fluid for people with type 2 spinal muscular atrophy (SMA). It may help improve motor function.
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