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4 recruiting studies matching your filters
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
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
Estimated
~$10 – $7,500
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