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RECRUITINGOBSERVATIONAL

Establishing Walking-related Digital Biomarkers in Rare Childhood Onset Progressive Neuromuscular Disorders

Important: This information is not medical advice. Talk to your doctor about whether a clinical trial is right for you.

About This Trial

The purpose of this research is (1) to identify disease specific walking-related digital biomarkers of disease severity, and (2) monitor longitudinal changes in natural environments, for extended periods of time, in DMD and SMA.

Who May Be Eligible (Plain English)

Who May Qualify: - Genetic confirmation of disease (DMD, SMA) or healthy control - Able to walk independently at least 25 meters - Ongoing corticosteroids therapy or initiation of corticosteroid therapy in the previous 3 months for DMD - Stable dose of FDA approved SMN up-regulator therapy or in an open-label extension phase of a study treatment for at least 6 months for SMA or gene replacement at enrollment for SMA or DMD participants. Who Should NOT Join This Trial: - Use foot orthoses or assistive devices for community ambulation or a mobility device for community navigation - Use investigational medications intended for treatment of NMD within 30 days - Prior to study entry had an injury or surgery that would impact gait within the previous 3 months Always talk to your doctor about whether this trial is right for you.

Original Eligibility Criteria

View original clinical language
Inclusion Criteria: * Genetic confirmation of disease (DMD, SMA) or healthy control * Able to walk independently at least 25 meters * Ongoing corticosteroids therapy or initiation of corticosteroid therapy in the previous 3 months for DMD * Stable dose of FDA approved SMN up-regulator therapy or in an open-label extension phase of a study treatment for at least 6 months for SMA or gene replacement at enrollment for SMA or DMD participants. Exclusion Criteria: * Use foot orthoses or assistive devices for community ambulation or a mobility device for community navigation * Use investigational medications intended for treatment of NMD within 30 days * Prior to study entry had an injury or surgery that would impact gait within the previous 3 months

Locations (3)

Stanford University
Palo Alto, California, United States
Boston Children's Hospital
Boston, Massachusetts, United States
Columbia University Irving Medical Center
New York, New York, United States