NEUROMUSCULAR DISEASES

This study is testing whether a new drug called taldefgrobep alfa can improve muscle strength and function in people with spinal muscular atrophy (SMA). The drug is designed to block a protein that limits muscle growth. Participants will be people already receiving standard SMA t…

This Phase 3 trial is testing whether a single dose of an experimental gene therapy called NTLA-2001 can safely slow or stop the progression of hereditary amyloidosis with polyneuropathy (ATTRv-PN), a rare genetic disease that damages nerves. About 50 participants worldwide will …

This study is testing whether a cell therapy called deramiocel can slow the loss of muscle function in boys and young men with Duchenne muscular dystrophy (DMD). About 106 participants will receive either the therapy or a placebo via IV infusion every three months for a year, wit…

This study aims to see if full-body muscle MRI scans can become a better, non-invasive tool for diagnosing rare neuromuscular diseases. Researchers will collect and analyze MRI scans from 1,000 patients across 14 international centers to identify unique patterns of muscle damage …

This study is testing a new, comfortable headset designed to help people with advanced ALS communicate. The device reads brain signals to let users type on a virtual keyboard without moving. Researchers are working with 5 participants to see how easy the headset is to set up, how…