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A Prospective Natural History and Outcome Measure Discovery Study of Charcot-Marie-Tooth Disease, Type 4J
This is a multicenter, longitudinal, prospective observational natural history study of subjects with a molecularly confirmed diagnosis of CMT4J. The study wil…
Center for Inherited Muscle Research Neuromuscular Research Biobank
The purpose of this research repository is to collect, store, and share with other researchers any tissues that subjects with all types of neuromuscular diseas…
Non-Invasive Respiratory Monitor
This study will clinically evaluate a newly developed respiratory monitor - pneuRIP. The pneuRIP uses Respiratory Inductance Plethysmography (RIP) bands to mea…
Non-invasive Brain-computer Interfaces for Control of Assistive Devices
Injuries affecting the central nervous system may disrupt the cortical pathways to muscles causing loss of motor control. Nevertheless, the brain still exhibit…
Measuring Physical Activity Levels and Intensity Using an ActivPal™ Accelerometer in Adult and Pediatric Populations With Neuromuscular Diseases
This study measures the level of physical activity in participants with neuromuscular disorders. The patient wears a small button like meter on their leg for a…
A Phase 1 Study of Anitocabtagene Autoleucel for the Treatment of Subjects With Non-oncology Plasma Cell-related Diseases
A Phase 1 dose-escalation study designed to evaluate the safety, tolerability, and preliminary efficacy of anito-cel in subjects with generalized myasthenia gr…