Congenital Myasthenic Syndrome Clinical Trial
Official title:
Multicenter, Multinational, Natural History Study in Participants With DOK7 Congenital Myasthenic Syndromes
Participants will attend up to 3 study visits to collect clinical assessments. The assessments will evaluate participants' symptoms and quality of life to understand disease activity in patients with DOK7-CMS better and may inform future study design.
n/a
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