A Prospective Natural History and Outcome Measure Validation Study of Congenital Myasthenic Syndromes
Recruiting
Observational Study
Myasthenic Syndromes, Congenital
No Placebo Group
Every participant receives an active treatment — no one gets a placebo.
No Study Drug
Researchers observe your health over time — no experimental treatment is given.
At a Glance
- Age
- 6 Months – 99
- Sex
- Any
- Study type
- Observational
- Participants needed
- 75 (estimated)
- Sponsor
- National Institute of Neurological Disorders and Stroke (NINDS) · NIH
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About This Trial
Background:
Congenital myasthenic syndromes (CMSs) are a group of inherited disorders that affect how the nerves communicate with muscles. These can cause many problems that affect how people can move and use their bodies.
Objective:
This is a natural history study to learn more about how CMSs affect the body and cause changes over time.
Eligibility:
People aged 6 months or older with a CMS. …
Trial Locations
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Eligibility Criteria
* INCLUSION CRITERIA:
In order to be eligible to participate in this study, an individual must meet all of the following criteria:
* Stated willingness to comply with all study procedures and availability for the duration of the study
* Male or female, aged \>= 6 months of age
* Clinically stable …
Contacts