- 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 - ongoing, recruitingTherapeutic use (Phase IV)
A Phase 4 Study of Nusinersen (BIIB058) Among Patients With Spinal Muscular Atrophy Who Received Onasemnogene Abeparvovec - RESPOND
This study looks at how well the medicine Spinraza (nusinersen) works for people with Spinal Muscular Atrophy (SMA) who have previously received another treatment called Zolgensma (onasemnogene abeparvovec). Researchers want to see if Spinraza can help improve movement and reduce problems for these patients.
Spain · Germany · Italy - active not recruitingNA
Acceptability, Feasibility, Safety and Efficacy of a Optimized Rehabilitation Program for Treated Patients With Spinal Muscular Atrophy (SMA).
This study looks at a new and improved rehabilitation program for children aged 1-10 with Spinal Muscular Atrophy (SMA) in the UK. It aims to see if more frequent, hands-on therapy and home equipment can help children and be a good option for families.
United Kingdom - 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 - recruitingNA
Study of an Intrathecal Port and Catheter System for Subjects With Spinal Muscular Atrophy
This study is testing a new device, the ThecaFlex DRx System, for people with Spinal Muscular Atrophy (SMA) to make it easier to give them their medicine. It involves having the device implanted and then being monitored for a year.
United States · Germany · Poland - 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 - 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