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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.

Treatment 321
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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.

Disease 244
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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.

Disease 255
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Combo Drug Treatment More Effective For Kidney Disease in Type 2 Diabetics

Drugs.com

MONDAY, June 9, 2025 — Prompt treatment with a drug combo can effectively manage chronic kidney disease in people with type 2 diabetes, a new clinical trial shows. Patients prescribed the combination of finerenone and empagliflozin.

Treatment 227
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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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Diabetes Drug 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 c.

Disease 244
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Lilly to acquire Verve in $1B bet on gene editing for heart disease

BioPharma Drive: Drug Pricing

The deal centers around a medicine that could “shift the treatment paradigm for cardiovascular disease,” Lilly said, but also follows a yearslong in decline in Verve’s share price amid skepticism over the need for such a therapy.

Disease 187