NEUROMUSCULAR DISEASE
Clinical trials for NEUROMUSCULAR DISEASE explained in plain language.
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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 DISEASE
Phase: PHASE3 • Sponsor: Intellia Therapeutics • Aim: Disease control
Last updated Mar 31, 2026 12:12 UTC
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Hunt for clues: major study aims to solve mysteries of rare muscle and nerve diseases
Knowledge-focused ENROLLING_BY_INVITATIONThis study aims to improve the diagnosis and understanding of various rare neuromuscular diseases, which affect muscles and nerves. Researchers will collect blood, tissue samples, and medical data from 500 adults with these conditions. The goal is to find genetic markers and othe…
Matched conditions: NEUROMUSCULAR DISEASE
Sponsor: Ottawa Hospital Research Institute • Aim: Knowledge-focused
Last updated Apr 02, 2026 02:11 UTC