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Q&A: One scientist’s bold vision to make on-demand treatments routine for life-threatening rare genetic diseases

Broad Institute

Based on a technology developed by Broad Institute core member David Liu’s laboratory, the treatment is the first in a series of new medicines being tested to treat rare diseases by repairing patients’ particular genetic misspellings. s to be produced at scale, making them the standard of care for life-threatening rare genetic diseases.

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Merck’s ENFLONSIA Receives ACIP Recommendation for Protecting Infants from Severe RSV

The Pharma Data

Merck’s ENFLONSIA Receives ACIP Endorsement for RSV Prevention in Infants, Marking a Major Milestone in Pediatric Infectious Disease Prevention U.S. According to CDC estimates, RSV leads to 58,000–80,000 hospitalizations and 100–300 deaths annually among children under five in the United States. pediatric population. Dr. Richard M.

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Sanofi Speeds Up Beyfortus Shipments Ahead of 2025-2026 RSV Season

The Pharma Data

According to Thomas Triomphe, Executive Vice President of Vaccines at Sanofi, the product has been administered to over 250,000 infants in real-world settings, backed by more than 40 studies demonstrating its effectiveness in preventing RSV lower respiratory tract disease.

Virus 40
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The Ongoing Challenges of Understanding Long COVID and Exploring Innovative Solutions

Perficient: Drug Development

Current Treatment Landscape and Therapeutic Approaches The management of Long COVID remains challenging due to its heterogeneous presentation, unclear pathophysiology, and the absence of approved disease-modifying therapies. Current approaches focus on symptom management, rehabilitation, and investigating potential underlying mechanisms.

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The Uncertain Origins of Aspirin

Codon

And some of the remedies are still practiced today: the treatment for Guinea-worm disease was, and still is , to wrap the emerging end of the worm around a stick and slowly pull it out. estimated to be about 29 million people) took daily aspirin to prevent cardiovascular disease. As a piece of history, it’s fantastic.

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Biocryst sells Europe business; Peter Marks criticizes new FDA vaccine rules

BioPharma Drive: Drug Pricing

Deep Dive Library Events Press Releases Topics Sign up Search Sign up Search Pharma Biotech FDA Clinical Trials Deals Drug Pricing Gene Therapy An article from News roundup Biocryst sells Europe business; Peter Marks criticizes new FDA vaccine rules Biocryst is offloading part of its business to an Italian drugmaker for $250 million upfront.

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Price of World’s First Malaria Vaccine for Children in Endemic Countries Slashed by Over 50%, Now Under $5

The Pharma Data

This announcement was made as part of their pledges to Gavi, the Vaccine Alliance, ahead of Gavi’s sixth replenishment cycle (Gavi 6.0) In a landmark move, Bharat Biotech will progressively reduce the price of the RTS,S vaccine to below $5 per dose by 2028—a reduction of more than 50%. scheduled for 2026 to 2030.

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