- 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 - 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