Spinal Muscular Atrophy (SMA) research hub
9 indexed studies · 6 currently recruiting
- recruitingTherapeutic exploratory (Phase II)
An Open-Label Study to Investigate the Safety, Tolerability, and Pharmacokinetics/Pharmacodynamics of Risdiplam (RO7034067) in Adult and Pediatric patients with Spinal Muscular Atrophy
This study is looking at a new medicine called Risdiplam for people with Spinal Muscular Atrophy (SMA). We want to understand how safe it is, if it causes side effects, and how the body handles the medicine. It's for both adults and children.
Netherlands · Italy · France - recruitingTherapeutic confirmatory (Phase III)
Longitudinal study of clinical outcome measures in adult patients with Spinal Muscular Atrophy (SMA) treated with Risdiplam.
This study looks at how a medicine called Risdiplam (also known as Evrysdi) helps adults with Spinal Muscular Atrophy (SMA) type 2 or 3. Doctors want to see if it improves their daily life and how their bodies are working over two years. It's a big step to understand this treatment better.
Belgium - active not recruitingNA
VRehab-SMA Phase 1.2
This study is looking into a new virtual reality game to help young people with Spinal Muscular Atrophy (SMA) with their exercises at home. It aims to make rehabilitation more fun and easier to access, especially for those who struggle to get regular physical therapy.
United Kingdom - active not recruitingPHASE2, PHASE3
A Study to Investigate the Safety and Efficacy of RO7204239 in Combination With Risdiplam (RO7034067) in Participants With Spinal Muscular Atrophy
This study is looking into a new combination of medicines, RO7204239 and risdiplam, for people with spinal muscular atrophy (SMA). The aim is to see if this combination is safe and helps improve muscle strength and movement.
United States · Australia · Belgium - enrolling by invitationNA
The Use of Assistive Gait Devices Can Reduce the Risk of Falls in Patients With Neuromuscular Diseases Following a Training Period.
This study looks at how walking aids and training can help people with certain muscle and nerve conditions fall less often. It aims to improve their safety and daily life by finding the best ways to use these aids.
Germany - recruitingPHASE2
A Study to Assess the Safety, Tolerability, Efficacy, Pharmacokinetics, and Immunogenicity of Intravenous Administration of ARGX-119 in Pediatric Participants Aged 5 to Less Than 18 Years With Spinal Muscular Atrophy
This study is looking at a new medicine called ARGX-119 for children aged 5 to 17 with Spinal Muscular Atrophy (SMA). It aims to find the right dose, check how safe it is, and see how well it works alongside their current SMA treatments to help muscle strength.
United States · Australia · Canada - recruiting
An International Federated Model for Wearable-derived Remote Longitudinal Motor Monitoring in Young Children With Spinal Muscular Atrophy Compared With Healthy Controls: Active-NBS Study (UK)
This study looks at how children with Spinal Muscular Atrophy (SMA) develop muscle movement compared to healthy children. Researchers use special wearable devices at home to track progress, hoping to find the best time to give new treatments to children with SMA.
United Kingdom - active not recruitingPHASE3
Long-term Follow-up of Patients With Spinal Muscular Atrophy Treated With OAV101 in Clinical Trials
This study looks at the long-term safety and effects of the medicine onasemnogene abeparvovec (OAV101) for Spinal Muscular Atrophy (SMA). It follows people who previously took part in an OAV101 trial for five years to see how they are doing.
United States · Australia · Belgium - recruiting
Adult SMA Research and Clinical Hub
This study, called Adult SMA REACH, collects information on adults with Spinal Muscular Atrophy (SMA) to understand how different treatments and care affect their lives. It aims to improve care and inform future research.
United Kingdom