Establishing Walking-related Digital Biomarkers in Rare Childhood Onset Progressive Neuromuscular Disorders
Recruiting
Observational Study
Spinal Muscular Atrophy Type 3
Duchenne Muscular Dystrophy (DMD)
No Placebo Group
Every participant receives an active treatment — no one gets a placebo.
Healthy Volunteers Welcome
You do not need to have the condition being studied to take part.
No Study Drug
Researchers observe your health over time — no experimental treatment is given.
At a Glance
- Age
- 5 and older
- Sex
- Any
- Study type
- Observational
- Participants needed
- 106 (estimated)
- Sponsor
- Columbia University · Other
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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.
Trial Locations
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Eligibility Criteria
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…
Contacts