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RECRUITING
Updated: Feb 5, 2026

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

Brief Summary

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.

Key Information

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

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