- RecruitingPHASE1
A Study to Find Out How Nusinersen is Processed in the Body When Given Through the ThecaFlex DRx™ System in Adult and Pediatric Participants With Spinal Muscular Atrophy (PIERRE-PK)
This study looks at how a medicine (nusinersen) for Spinal Muscular Atrophy (SMA) travels through the body when given in two ways: a standard spinal injection or using a new implantable system called ThecaFlex DRx. It aims to see which method delivers the medicine best.
For: Muscular Atrophy, SpinalUnited States · France · Germany - 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 - Active not recruitingPHASE3
A Study to Learn About the Effect of Higher Doses of Nusinersen (BIIB058) Given as Injections to Participants With Spinal Muscular Atrophy (SMA) Who Were Previously Treated With Risdiplam (ASCEND)
This study looks at a higher dose of nusinersen for teenagers and adults with Spinal Muscular Atrophy (SMA) who can't walk and have previously taken risdiplam. Researchers want to see if this higher dose helps improve muscle and movement abilities, especially in the arms and hands, and to check its safety.
For: Spinal Muscular AtrophyUnited States · Germany · Hungary