NEUROMUSCULAR DISEASES (NMD)
Clinical trials for NEUROMUSCULAR DISEASES (NMD) explained in plain language.
Never miss a new study
Get alerted when new NEUROMUSCULAR DISEASES (NMD) trials appear
Sign up with your email to follow new studies for NEUROMUSCULAR DISEASES (NMD), keep track of the ones that matter, and come back to a personal dashboard instead of checking manually.
By submitting, you agree to our Terms of use
-
One-Shot gene therapy aims to halt rare, devastating nerve disease
Disease control OngoingThis 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 …
Matched conditions: NEUROMUSCULAR DISEASES (NMD)
Phase: PHASE3 • Sponsor: Intellia Therapeutics • Aim: Disease control
Last updated Mar 31, 2026 12:12 UTC
-
One patient, one drug: custom gene therapy trial launches for Ultra-Rare disease
Disease control OngoingThis is a highly personalized study for a single patient with a rare genetic nerve disease called CMT2S. The trial is testing the safety of a custom-made drug, VCA-894A, designed to fix a specific genetic error. The drug is injected into the spinal fluid to try to restore a missi…
Matched conditions: NEUROMUSCULAR DISEASES (NMD)
Phase: PHASE1, PHASE2 • Sponsor: Vanda Pharmaceuticals • Aim: Disease control
Last updated Mar 31, 2026 12:11 UTC