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

~Estimated

~$10 – $1,125

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

~Estimated

~$10 – $8,750

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)

~Estimated

~$10 – $750

Columbia UniversityNCT06839469

Phase 2

A Phase II, Open-label Study to Investigate the Pharmacokinetics and Safety of Risdiplam in Infants With Spinal Muscular Atrophy

This study will evaluate the pharmacokinetics (PK) and safety of risdiplam in participants with spinal muscular atrophy (SMA) under 20 days of age at first dos…

Muscular Atrophy, Spinal

~Estimated

~$10 – $7,500

Hoffmann-La RocheNCT05808764

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