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Identification of Gait-related Digital Biomarkers of Bone Health in Spinal Muscular Atrophy
The objective of this study is to understand how spatiotemporal and kinetic gait parameters are associated with bone health in pwSMA who are receiving DMT. We …
Spinal Muscular Atrophy Type 3Ambulatory Spinal Muscular Atrophy
Jacqueline MontesNCT07400198
Phase 2
A Phase 2, Double-Blind Study to Evaluate the Pharmacokinetics, Pharmacodynamics, Efficacy, and Safety of Apitegromab in Subjects <2 Years Old With Spinal Muscular Atrophy (SMA)
This double-blind, Phase 2, multiple-dose study will be conducted to evaluate the PK/PD, efficacy, safety, and tolerability of apitegromab in subjects \<2 year…
Spinal Muscular AtrophySMASpinal Muscular Atrophy Type 2+3 more
Scholar Rock, Inc.NCT07047144
Establishing Walking-related Digital Biomarkers in Rare Childhood Onset Progressive Neuromuscular Disorders
The purpose of this research is (1) to identify disease specific walking-related digital biomarkers of disease severity, and (2) monitor longitudinal changes i…
Spinal Muscular Atrophy Type 3Duchenne Muscular Dystrophy (DMD)
Columbia UniversityNCT06839469