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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
The Foundation for Orthopaedics and Regenerative MedicineNCT07226726
Phase 1
A Phase 1b, Double-Blinded, Randomized, Placebo-Controlled Study to Assess the Safety, Tolerability, Pharmacokinetics, Immunogenicity, and Efficacy of ARGX-119 in Adult Participants With DOK7-Congenital Myasthenic Syndromes
The purpose of this study is to assess the safety and tolerability of ARGX-119 in adult participants with DOK7- Congenital Myasthenic Syndromes. The study will…
Congenital Myasthenic SyndromeCMS
argenxNCT06436742
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
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
argenxNCT06078553