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Benzinga
Benzinga
Business
Vandana Singh

uniQure's Investigational Gene Therapy Shows Promising Efficacy With Manageable Safety Risks For Fabry Disease

QURE uniQure Disease Gene Therapy
gene therapy

uniQure N.V. (NASDAQ:QURE) on Friday released initial safety and exploratory efficacy data from the first cohort of its Phase 1/2a trial of AMT-191, an investigational gene therapy for Fabry disease.

Fabry disease is a genetic lysosomal storage disorder characterized by a deficiency of the enzyme that breaks down fats.

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