Gene-Editing Therapy Promising for Rare Hereditary Disease



(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…



Source link : https://www.medpagetoday.com/neurology/generalneurology/117732

Author :

Publish date : 2025-10-01 15:23:00

Copyright for syndicated content belongs to the linked Source.

Related Content

Trial Makes the Case for Treating Knee Osteoarthritis With Radiation

By News Health October 1, 2025

RFK Jr.'s Text to Tylenol's Maker; CDC's Anti-Vax Hire; NIH Spends All Its Money

By News Health October 1, 2025

What Are the Goalposts for Present and Future ATTR-CM Therapies?

By News Health October 1, 2025

Morphine No Help for Chronic Breathlessness From Cardiorespiratory Conditions

By News Health October 1, 2025

Autism may have subtypes that are genetically distinct from each other

By News Health October 1, 2025

COVID Vaccines and Cancer Risk: Experts Dissect Flawed Study

By News Health October 1, 2025
Exit mobile version