Nemaline myopathy
MONDO:0018958Nemaline myopathy (NM) encompasses a large spectrum of myopathies characterized by hypotonia, weakness and depressed or absent deep tendon reflexes, with pathologic evidence of nemaline bodies (rods) on muscle biopsy.
Also known as: NEM, NM, nemaline body disease, nemaline myopathy, nemaline rod myopathy, rod myopathy, Rod body disease, Rod-body myopathy
17 clinical trials for this condition and its sub-types.
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Broader categories
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One-Shot gene therapy could end monthly eye injections for wet AMD
Disease control OngoingThis study tests a one-time gene therapy called RGX-314 for wet age-related macular degeneration (AMD), a leading cause of vision loss. About 714 adults aged 50-89 who currently need regular eye injections will receive either the gene therapy or standard treatment. The goal is to…
Phase: PHASE3 • Sponsor: AbbVie • Aim: Disease control
Last updated Aug 05, 2026 00:00 UTC
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Gene therapy could end frequent eye injections for wet AMD
Disease control OngoingThis study tests a one-time gene therapy called RGX-314 for wet age-related macular degeneration (AMD), a leading cause of vision loss. The treatment aims to help the eye produce its own medicine to stop leaky blood vessels, potentially replacing the need for repeated eye injecti…
Phase: PHASE2, PHASE3 • Sponsor: AbbVie • Aim: Disease control
Last updated Jun 27, 2026 12:24 UTC
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UK study tracks rare muscle disease to pave way for future treatments
Knowledge-focused OngoingThis study follows 45 people with nemaline myopathy in the UK to learn how the disease naturally progresses. Researchers collect medical exams, quality-of-life surveys, and physical assessments over time. The goal is to identify the best ways to measure the disease in future clin…
Sponsor: University of Oxford • Aim: Knowledge-focused
Last updated Jun 27, 2026 12:07 UTC