Observation Study in Patients Age 0-5 Years With LAMA2-related Congenital Muscular Dystrophy

Recruiting Observational Study
LAMA2-MD \(Merosin Deficient Congenital Muscular Dystrophy, MDC1A\)
No Study Drug Researchers observe your health over time — no experimental treatment is given.
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At a Glance
Age
Up to 5
Sex
Any
Study type
Observational
Participants needed
44 (estimated)
Sponsor
Nationwide Children's Hospital · Other
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About This Trial

The goal of this observational study is to understand how young children with LAMA2-related dystrophy move and change over time. We will also learn about how this condition impacts other body systems. Participants will undergo: * Neuromuscular assessments * Blood collections * Swallowing and breathing assessments * Questionnaires

Trial Locations
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Eligibility Criteria
Inclusion Criteria: * Signed informed consent by the subject, parent(s) or legally authorized representative (LAR) and/or assent by the subject (when applicable). * Subject must be aged birth to less than 5.0 years of age at time of consent. * A confirmed diagnosis of LAMA2-RD confirmed via: a: …
Contacts

Kevin Warf

(614) 355-2765

ReadyCMDLAMA2@nationwidechildrens.org

CONTACT