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

Science Daily: Pharmacology News

The infant, who was diagnosed with the rare condition carbamoyl phosphate synthetase 1 (CPS1) deficiency shortly after birth, has responded positively to the treatment. The process, from diagnosis to treatment, took only six months and marks the first time the technology has been successfully deployed to treat a human patient.

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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.

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New tRNA tech aims to rewrite rare disease treatment

Drug Target Review

Alltrna’s unique approach focuses on addressing genetic diseases at a fundamental level, shifting the paradigm from a one-disease, one-therapy model to a mutation-specific strategy that could transform the treatment of hundreds of rare diseases. This will mark a critical step in validating the potential of engineered tRNA therapeutics.

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Nanorobot hand made of DNA grabs viruses for diagnostics and blocks cell entry

Science Daily: Pharmacology News

Dubbed the NanoGripper, the nanorobotic hand also could be programmed to interact with other viruses or to recognize cell surface markers for targeted drug delivery, such as for cancer treatment.

DNA
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Bridging Innovation & Patient Care: The Growing Role of AI

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

From Research to Real-World Care 🌎 Understand how AI-driven advancements are shaping personalized treatments and patient outcomes. Key Takeaways: Faster, Smarter Trials ⚡ Learn how AI is streamlining patient recruitment, data collection, and trial efficiency. Register today to save your seat!

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Discovery of IHMT-15130 as a Highly Potent Irreversible BMX Inhibitor for the Treatment of Myocardial Hypertrophy and Remodeling

Covalent Modifiers

This study provides further experimental evidence for the application of BMX kinase inhibitors in the treatment of cardiac hypertrophy.

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Novel stem cell therapy repairs 'irreversible' corneal damage in clinical trial

Science Daily: Pharmacology News

Results from a phase 1/2 clinical trial of a novel stem cell treatment for cornea injuries found 14 patients treated and tracked for 18 months had a more than 90% success rate at restoring the cornea's surface and improvements in vision.

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From Diagnosis to Delivery: How AI is Revolutionizing the Patient Experience

Speaker: Simran Kaur, Founder & CEO at Tattva Health Inc.

Key Learning Objectives: Enhance Patient Communication 📲 Explore how AI facilitates more personalized, timely, and efficient patient interactions, improving overall engagement and satisfaction Leverage AI for Improved Patient Care & Predictive Medicine 🧬 Learn how AI helps healthcare providers offer more precise, preventive care by (..)