Spinal Muscular Atrophy Type I research hub
6 indexed studies · 3 currently recruiting
- active not recruitingPHASE3
Long-term Follow-up Study of Patients Receiving Onasemnogene Abeparvovec-xioi
This study is checking on people with Spinal Muscular Atrophy (SMA) who have already had a special gene treatment called onasemnogene abeparvovec-xioi. We want to see how they are doing in the long term, both with their health and how well the treatment keeps working.
United States · Australia · Belgium - recruiting
Study of the Reproducibility of the French Version of the Modified SMAFRS Scale
This study is checking if a French version of a questionnaire for people with Spinal Muscular Atrophy (SMA) gives consistent results. It helps doctors understand how SMA affects daily life, like eating and walking. Participants will fill out the questionnaire twice to see if it's reliable.
France - recruiting
UK SMA Patient Registry
This registry is for people with Spinal Muscular Atrophy (SMA) in the UK and Ireland. It gathers information online to help doctors and researchers better understand SMA, which is a condition that affects how muscles work. You can share details about your health and daily life.
United Kingdom - active not recruitingPHASE3
Long-Term Safety & Efficacy of Apitegromab in Patients With SMA Who Completed Previous Trials of Apitegromab
This study looks at the long-term safety and effects of a medicine called apitegromab for people with Spinal Muscular Atrophy (SMA) types 2 and 3. It's for those who have already completed earlier studies with this drug.
United States · Belgium · France - recruitingPHASE2
A Study to Evaluate How Apitegromab Works in Subjects Who Are Less Than 2 Years Old and Have Spinal Muscular Atrophy
This study is looking at a new medicine called apitegromab for babies and toddlers under 2 years old who have a condition called spinal muscular atrophy (SMA). It aims to see how safe and effective the medicine is, how the body uses it, and if it helps with movement.
United States · Belgium · France - active not recruitingPHASE3
A Study to Evaluate the Efficacy and Safety of Taldefgrobep Alfa in Participants With Spinal Muscular Atrophy
This study looks at a new medicine called taldefgrobep alfa for young people with Spinal Muscular Atrophy (SMA). It's for those already on other SMA treatments to see if adding this new medicine can further improve muscle strength and movement.
United States · Belgium · Czechia