Spinal muscular atrophy gene study
Treatments studied
Part of Brain & nervous system clinical trials.
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.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You are an adult (18 or older).
- You have either 1 or 3 copies of the SMN1 gene (control group), or 2 copies of the SMN1 gene in a special pattern called '2+0' (test group).
- You have French health insurance.
- You are able to sign a consent form.
- You are not pregnant or breastfeeding.
- You are not in prison, under guardianship, or under legal protection.
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 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 study uses wearable devices (like smartwatches or activity trackers) to monitor movement and motor development in young children with spinal muscular atrophy (SMA), a rare genetic condition affecting muscle strength. The goal is to see if wearables can track disease progression and treatment response better than traditional clinic visits.
This study follows people with spinal muscular atrophy (SMA) who have a particular genetic change in the SMN1 gene. It compares long-term outcomes for those who do and do not receive disease-modifying treatments, to better understand what helps over time.
This study is looking for children and teens with spinal muscular atrophy (SMA) who are already on a treatment like Spinraza, Evrysdi, or Zolgensma. The goal is to understand different forms of SMA and how the treatment affects them over time.
This is a long-term observation study that follows people affected by spinal muscular atrophy (SMA), people who carry the SMA gene but have no symptoms, and families of people with SMA. It helps researchers better understand SMA and related genetic status, which can support future treatment planning.
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