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A Natural History Study in Participants With Congenital Myasthenic Syndromes (CMS) Due to Mutations in DOK7, MUSK, AGRN, or LRP4
Sponsor: argenx
Summary
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
Official title: Multicenter, Multinational, Natural History Study in Participants With Congenital Myasthenic Syndromes Due to Mutations in DOK7, MUSK, AGRN, or LRP4
Key Details
Gender
All
Age Range
2 Years - Any
Study Type
OBSERVATIONAL
Enrollment
100
Start Date
2024-02-13
Completion Date
2027-06
Last Updated
2026-03-13
Healthy Volunteers
No
Conditions
Locations (19)
UC Davis Health - UC Davis Health Midtown Ambulatory Care Center
Sacramento, California, United States
Ann and Robert H Lurie Children's Hospital of Chicago - Main Hospital
Chicago, Illinois, United States
Hospital Sisters Health System (HSHS) - St Elizabeth's Hospital
O'Fallon, Illinois, United States
Medical University of Vienna
Vienna, Austria
The Ottawa Hospital - Civic Campus
Ottawa, Canada
CHU Bordeaux - Groupe Hospitalier Pellegrin - Neurology
Bordeaux, France
Hospices Civils de Lyon (HCL) - Hopital Pierre Wertheimer - Neurology
Bron, France
CHU Lille Hopital Salengro - Neurology
Lille, France
CHU Timone
Marseille, France
Assistance Publique Hopitaux de Paris - Hopital Pitie-Salpetriere
Paris, France
Universitaetsklinikum Essen (AoR)
Essen, Germany
Fondazione IRCCS Istituto Neurologico Carlo Besta
Milan, Italy
National Hospital Organization Akita Hospital - Neurology
Yurihonjō, Japan
Centrum Medyczne Neurologia Slaska - Neurology
Katowice, Poland
Hospital Sant Joan de Deu - Pediatric Neurology
Esplugues de Llobregat, Spain
Hospital Universitario 12 de Octubre
Madrid, Spain
Hospital Universitari i Politecnic La Fe
Valencia, Spain
Ulster Hospital
Dundonald, United Kingdom
Oxford University Hospitals NHS Foundation Trust - John Radcliffe Hospital
Oxford, United Kingdom