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
Can understand the requirements of the study and can provide written informed consent/assent, and willingness and ability to comply with the study protocol procedures
Is male or female and aged ≥2 years at the time of providing informed consent/assent
Has a diagnosis of CMS due to biallelic pathogenic mutations in DOK7 or any pathogenic mutations in MUSK, AGRN, or LRP4
Has a total Quantitative Myasthenia Gravis (QMG) score of ≥3 (applies only to participants aged ≥6 years)
Known medical condition that would interfere with an accurate assessment of CMS, in the investigator's opinion
Is currently participating in any interventional clinical study with a study drug at the time of providing informed consent/assent
Diagnosis of CMS due to mutation of any gene other than DOK7, MUSK, AGRN, or LRP4