ClinicaliQ Trial Snapshot
- A Natural History Study in Participants With Congenital Myasthenic Syndromes (CMS) Due to Mutations in DOK7, MUSK, AGRN, or LRP4 — Recruiting • NCT06078553.
- Study tracking symptom changes and quality of life in patients with rare inherited muscle-nerve connection disorders caused by specific genetic mutations.
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What This Trial Is Studying
Participants will attend up to 4 study visits to collect clinical assessments. The assessments will evaluate participants' symptoms and quality of life to understand disease activity in patients with CMS due to mutations in DOK7, MUSK, AGRN, or LRP4. More information can be found here: https://clinicaltrials.argenx.com/cms Conditions: Congenital Myasthenic Syndrome Lead Sponsor: argenx Planned Enrollment: 100 participants