PERIPHERAL NERVOUS SYSTEM DISEASE
Clinical trials for PERIPHERAL NERVOUS SYSTEM DISEASE explained in plain language.
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One-shot gene therapy aims to stop rare nerve disease in its tracks
Disease control Recruiting nowThis study tests a single dose of a gene therapy called NTLA-2001 in 60 people with a rare inherited nerve disease (ATTRv-PN). The goal is to see if it can safely reduce harmful protein buildup and slow nerve damage compared to a placebo. Participants must have a confirmed diagno…
Matched conditions: PERIPHERAL NERVOUS SYSTEM DISEASE
Phase: PHASE3 • Sponsor: Intellia Therapeutics • Aim: Disease control
Last updated May 22, 2026 14:01 UTC
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Scientists launch massive study to unlock secrets of rare inherited nerve diseases
Knowledge-focused Recruiting nowThis study aims to learn more about rare inherited neurological disorders by observing up to 3,500 people with these conditions and their relatives. Researchers will collect medical history, perform exams, and take blood or skin samples to study genetics and disease progression. …
Matched conditions: PERIPHERAL NERVOUS SYSTEM DISEASE
Sponsor: National Institute of Neurological Disorders and Stroke (NINDS) • Aim: Knowledge-focused
Last updated May 26, 2026 07:50 UTC