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3 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
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