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Gene-Editing Therapy Promising for Rare Hereditary Disease

Summary by MedPage Today
(MedPage Today) -- A novel treatment based on CRISPR gene editing showed benefits in hereditary transthyretin amyloidosis with polyneuropathy (ATTRv-PN) in an early study. Among 36 patients who received a one-time dose of investigational nexiguran...

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MedPage Today broke the news in New York, United States on Wednesday, October 1, 2025.
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