A Natural History Study in Participants With Congenital Myasthenic Syndromes (CMS) Due to Mutations in DOK7, MUSK, AGRN, or LRP4
Start Date
2/13/2024
Completion Date
6/1/2027
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
Eligibility Criteria
Age Range: 2 years to No maximum
Conditions
Locations
UC Davis Health - UC Davis Health Midtown Ambulatory Care Center
Sacramento, California 95816
United States
Ann and Robert H Lurie Children's Hospital of Chicago - Main Hospital
Chicago, Illinois 60611
United States
Hospital Sisters Health System (HSHS) - St Elizabeth's Hospital
O'Fallon, Illinois 62269
United States
Harvard Medical School - Boston Children's Hospital
Boston, Massachusetts 02115
United States
Ohio State Martha Moorehouse
Columbus, Ohio 43221
United States
The Children's Hospital of Philadelphia (CHOP)
Philadelphia, Pennsylvania 19104
United States
Medical University of Vienna
Vienna, 1090
Austria
Hopital Universitaire de Bruxelles/ Academisch Ziekenhuis Brussel
Brussels, Anderlecht 1070
Belgium
Pseg Centro De Pesquisa ClinicaS.A
São Paulo, 04038-002
Brazil
Universidade de Sao Paulo (USP) Hospital das Clinicas da Faculdade de Medicina (HCFMUSP)
São Paulo, 05403-900
Brazil
The Children's Hospital of Eastern (CHEO), Ottawa
Ottawa, Ontario K1H8L1
Canada
The Ottawa Hospital - Civic Campus
Ottawa, K1Y 4E9
Canada
CHU Bordeaux - Groupe Hospitalier Pellegrin - Neurology
Bordeaux, 33076
France
Hospices Civils de Lyon (HCL) - Hopital Pierre Wertheimer - Neurology
Bron, 69500
France
CHU Lille Hopital Salengro - Neurology
Lille, 59037
France
CHU Timone
Marseille, 13385
France
Assistance Publique Hopitaux de Paris - Hopital Pitie-Salpetriere
Paris, 75013
France
Centre Hospitalier Regional Universitaire de Strasbourg - Hopital de Hautepierre
Strasbourg, 67000
France
Charite - Universitaetsmedizin Berlin
Berlin, 10117
Germany
UKRUB - Berufsgenossenschaftliches Universitatsklinikum Bergmannsheil GmbH
Bochum, 44789
Germany
Universitaetsklinikum Essen (AoR)
Essen, 45147
Germany
Fondazione IRCCS Istituto Neurologico Carlo Besta
Milan, 20133
Italy
National Center Hospital, National Center of Neurology and Psychiatry
Kodaira, 187-8551
Japan
National Hospital Organization Akita Hospital - Neurology
Yurihonjō, 018-1393
Japan
Medicover Integrated Clinical Services (MICS) Centrum Medyczne Bydgoszcz
Bydgoszcz, 85-065
Poland
Centrum Medyczne Neurologia Slaska - Neurology
Katowice, 40-689
Poland
Uniwersyteckie Centrum Kliniczne Warszawskiego Uniwersytetu Medycznego
Warsaw, 02-097
Poland
Hospital Sant Joan de Deu - Pediatric Neurology
Esplugues de Llobregat, 08950
Spain
Hospital Universitario 12 de Octubre
Madrid, 28041
Spain
Hospital Universitari i Politecnic La Fe
Valencia, 46026
Spain
Ulster Hospital
Dundonald, BT16 1RH
United Kingdom
Oxford University Hospitals NHS Foundation Trust - John Radcliffe Hospital
Oxford, OX3 9DU
United Kingdom