Browse Studies — Arctuva

Multicenter, Multinational, Natural History Study in Participants With Congenital Myasthenic Syndromes Due to Mutations in DOK7, MUSK, AGRN, or LRP4

Participants will attend up to 4 study visits to collect clinical assessments. The assessments will evaluate participants' symptoms and quality of life to unde…

Congenital Myasthenic Syndrome

Estimated

~$10 – $1,125

argenxNCT06078553

Phase 1

Mesenchymal Stem Cell Exosome Treatment of Congenital Myasthenic Syndrome

Patients with Congenital Myasthenic Syndrome will be treated with Mesenchymal Stem Cell Exosome solution.

Congenital Myasthenic Syndrome

Estimated

~$10 – $6,250

The Foundation for Orthopaedics and Regenerative MedicineNCT07226726

Congenital Muscle Disease Patient and Proxy Reported Outcome Study

The Congenital Muscle Disease Patient and Proxy Reported Outcome Study (CMDPROS) is a longitudinal 10 year study to identify and trend care parameters, adverse…

Congenital Muscular Dystrophy With ITGA7 (Integrin Alpha-7) DeficiencyAlpha-Dystroglycanopathy (Congenital Muscular Dystrophy and Abnormal Glycosylation of Dystroglycan With Severe Epilepsy)Alpha-Dystroglycanopathy (Congenital Muscular Dystrophy With Fatty Liver and Infantile-onset Cataract Caused by TRAPPC11 Mutations)+49 more