Antibody screening for gene therapy in neuromuscular disease
Part of Genetic & congenital clinical trials.
This study checks if you have antibodies that could affect gene therapy for neuromuscular diseases. It helps doctors know if gene therapy might work for you.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You are 6 years old or older, and under 60 years old.
- You have a genetic neuromuscular disease.
- You have not had gene or cell therapy before.
- You are covered by national health insurance.
- You (or your guardian) agree to take part.
Quick eligibility check
Answer a few plain-language questions, based on this study's own requirements, to get a preliminary sense of fit.
Similar studies
Other trials that look related to this one.
This trial tests a gene therapy for people with a specific mutation (S295L) in the SLC6A1 gene, which causes a neurodevelopmental disorder. The therapy aims to correct the genetic issue, and participants will be monitored closely for safety and effectiveness.
This early-phase study tests a gene therapy meant to treat people who have IGHMBP2 gene changes. It aims to improve the way the nervous system works and to see if the treatment is safe, especially in young children.
This study tests a new gene therapy called SNUG01 for people with ALS (a disease that weakens muscles). The goal is to see if it's safe and might slow down the disease, especially for those diagnosed within the last 2 years.
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 early-stage trial tests VGN-R09b, a gene therapy, given to children with AADC deficiency who are not doing well on standard medicines. It aims to improve symptoms and quality of life by addressing the underlying cause of AADC deficiency.
This trial tests a one-time gene therapy (SAR446268) for people with non-congenital myotonic dystrophy type 1. It aims to see if the treatment is safe and can improve muscle symptoms like stiffness and weakness.
Hear when a new Genetic Disorder trial opens
We’ll email you when one opens — at most once a week, no account needed, unsubscribe anytime.