- 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.
For: Spinal Muscular Atrophy · SMA · Spinal Muscular Atrophy Type 2United States · Belgium · France - 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.
For: Spinal Muscular Atrophy (SMA)United Kingdom - RecruitingPHASE4
A Study Evaluating the Effectiveness and Safety of Risdiplam Administered as an Early Intervention in Pediatric Participants With Spinal Muscular Atrophy After Gene Therapy
This study looks at giving Risdiplam to young children with Spinal Muscular Atrophy (SMA) who have already had gene therapy. It wants to see if this combination helps them even more and if it's safe. It's for children under 2 years old with a specific type of SMA.
For: Muscular Atrophy, SpinalUnited States · Germany · Poland - 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.
For: Spinal Muscular Atrophy (SMA)United States · Australia · Belgium - RecruitingPHASE2
A Study to Investigate the Pharmacokinetics and Safety of Risdiplam in Infants With Spinal Muscular Atrophy
This study is looking into a medicine called risdiplam for babies born with spinal muscular atrophy (SMA). It aims to understand how the medicine works in their bodies and if it's safe for very young infants (under 20 days old) at their first dose.
For: Muscular Atrophy, SpinalUnited States · Belgium · Canada - RecruitingPHASE4
A Study Evaluating the Effectiveness and Safety of Risdiplam Administered in Pediatric Patients With Spinal Muscular Atrophy Who Experienced a Plateau or Decline in Function After Gene Therapy
This study looks at a medicine called risdiplam for young children (under 2) with a muscle condition called spinal muscular atrophy (SMA). It's for those who had gene therapy but aren't getting better or are getting worse. The study checks if risdiplam is safe and helps them.
For: Muscular Atrophy, SpinalUnited States · Germany · Israel