Spinal Muscular Atrophy (SMA) research hub
6 indexed studies · 3 currently recruiting
- 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 - 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 - 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
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 - ongoing, 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 - ongoing, 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