Testing EXG001-307 for spinal muscular atrophy in infants
Part of Brain & nervous system clinical trials.
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.
Summary written for real people, not researchers, by Clin2.
Who can take part
- Your baby must have a confirmed diagnosis of SMA types 1 or 2.
- For type 1 SMA, the baby must be 6 months or younger on the day of treatment. For type 2 SMA, the baby must be between 6 months and 2 years old.
- Your baby must have a specific number of SMN2 genes: exactly 2 for type 1, or 3 or fewer for type 2.
- Your baby cannot have used any other SMA treatments like nusinersen or Zolgensma before.
- Your baby cannot need a breathing tube or use a breathing machine for 12 or more hours each day.
- Legal guardians must agree to follow all study visits and tests, and accept standard care like feeding tubes or breathing support if needed.
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 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.
This trial tests whether a non-invasive brain stimulation technique called transcranial magnetic stimulation (TMS) is safe and tolerable for people with spinal muscular atrophy (SMA). It may help improve muscle function by activating low motor neurons.
This study tests a new medicine called ARGX-119 in children with SMA. It is given through an IV and aims to help kids who can already walk get stronger and stay healthy.
This study looks at changes in two genes, SMN1 and SMN2, which are linked to spinal muscular atrophy (SMA). It aims to better understand the different versions of these genes in healthy people and those with SMA. Participating involves a blood sample and genetic analysis.
This study follows up on patients with type 1 spinal muscular atrophy (SMA) who already received the EXG001-307 gene therapy in a previous trial. The goal is to monitor their long-term health and safety over time.
This trial tests a new gene therapy called GCB-001 for children with type 2 spinal muscular atrophy who can sit but not walk. It aims to improve muscle function and overall health.
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