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Infant with rare, incurable disease is first to successfully receive personalized gene therapy treatment

Science Daily: Pharmacology News

A research team has developed and safely delivered a personalized gene editing therapy to treat an infant with a life-threatening, incurable genetic disease. The infant, who was diagnosed with the rare condition carbamoyl phosphate synthetase 1 (CPS1) deficiency shortly after birth, has responded positively to the treatment.

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Three-person DNA IVF stops inherited disease—eight healthy babies born in UK first

Science Daily: Pharmacology News

The process, known as pronuclear transfer, was designed to prevent the inheritance of devastating mitochondrial diseases passed down through the mother’s DNA. The early results are highly promising: all the babies are developing normally, and the disease-causing mutations are undetectable or present at levels too low to cause harm.

DNA
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Ultrafast 12-minute MRI maps brain chemistry to spot disease before symptoms

Science Daily: Pharmacology News

Illinois engineers fused ultrafast imaging with smart algorithms to peek at living brain chemistry, turning routine MRIs into metabolic microscopes. The system distinguishes healthy regions, grades tumors, and forecasts MS flare-ups long before structural MRI can. Precision-medicine neurology just moved closer to reality.

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GSK to buy liver disease drug for $1.2B

BioPharma Drive: Drug Pricing

The new acquisition from Boston Pharmaceuticals adds to a wave of dealmaking undertaken by GSK to build its liver disease franchise.

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Bridging Innovation & Patient Care: The Growing Role of AI

Speaker: Simran Kaur, Co-founder & CEO at Tattva.Health

Navigating the AI Landscape 🤖 Explore the opportunities and challenges with integrating AI in healthcare and disease prevention. From Research to Real-World Care 🌎 Understand how AI-driven advancements are shaping personalized treatments and patient outcomes. Register today to save your seat!

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Researchers devise new way to target and correct disease-related proteins

Broad Institute

Related groups Xavier lab Over the past two decades, large genetic studies have linked tens of thousands of DNA variants to thousands of human traits and diseases. Yet, correcting the effects of those variants to treat disease has been hampered in part by the lack of precise molecular tools to do so.

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Diabetes Drug, Dapagliflozin, Effective For Fatty Liver Disease

Drugs.com

FRIDAY, June 6, 2025 — A repurposed diabetes drug can be an effective tool against fatty liver disease, a new clinical trial has found. Dapagliflozin helped people reduce fat levels in their liver and avoid liver scarring that comes with the.