NEUROMUSCULAR DISEASES

This phase 3 trial tests whether a cell therapy called deramiocel (CAP-1002) can slow muscle decline in boys and young men with Duchenne muscular dystrophy (DMD). About 106 participants receive either the therapy or a placebo every 3 months for a year, then all can receive the th…

This study tests whether adding taldefgrobep alfa to standard SMA treatments can improve muscle function in people with spinal muscular atrophy. About 269 participants, both walkers and non-walkers, will receive either the drug or a placebo for 48 weeks. The main goal is to see i…

This study looks at whether using a machine that helps inflate the lungs (mechanical insufflation) twice a day can slow the loss of cough strength in people with early ALS. Twenty participants will use the BiWaze Cough device for 6 months. Their results will be compared to a simi…

This study is testing a new, comfortable headset that reads brain signals to help people with ALS communicate by typing on a virtual keyboard. The headset uses dry electrodes and is designed for daily use. Five people with ALS will try the device, and researchers will measure how…

This study aims to see if muscle MRI scans can accurately diagnose different types of neuromuscular diseases (muscle and nerve disorders). Researchers will collect MRI scans from 1000 patients across 14 centers to identify unique patterns of muscle damage. If successful, this non…