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Congenital Myasthenic Syndrome
Clinical trial pipeline · Data from ClinicalTrials.gov
See which Congenital Myasthenic Syndrome trials you may qualify forClinical trial pipeline · Data from ClinicalTrials.gov
See which Congenital Myasthenic Syndrome trials you may qualify forParticipants will attend up to 4 study visits to collect clinical assessments. The assessments will evaluate participants' symptoms and quality of life to under…
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…
This single-arm pilot study evaluates the effects of whole-body electrical muscle stimulation (WB-EMS) exercise on neuromuscular and physical function in adults…
Background: Congenital myasthenic syndromes (CMSs) are a group of inherited disorders that affect how the nerves communicate with muscles. These can cause many…
Patients with Congenital Myasthenic Syndrome will be treated with Mesenchymal Stem Cell Exosome solution.
The ActiLiège-Adult study is a prospective, longitudinal, observational study designed to collect natural history data on adult patients with neurological or me…
Currently, there are no standardised fall risk scores or guidelines on when to use appropriate assistive gait devices (AGDs) for people with neuromuscular disor…
Patients with ALS and other Motor Discorders will be treated with mesenchymal cell exosome solution.
Newborn screening (NBS) is a global initiative of systematic testing at birth to identify babies with pre-defined severe but treatable conditions. With a simple…