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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. Some of these treatments, like K.J.’s, The team that treated K.J. Today, K.J.

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

The Pharma Data

CDC panel backs long-acting monoclonal antibody to protect infants during their first RSV season; drug also included in federal Vaccines for Children program Merck , operating as MSD outside the United States and Canada, has announced a key regulatory milestone for its respiratory syncytial virus (RSV) prevention program. What Is ENFLONSIA™?

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FDA takes major step to ease access to CAR-T therapy

BioPharma Drive: Drug Pricing

Alamy Dive Brief: The Food and Drug Administration is easing limitations it imposed around the complex cancer drugs known as CAR-T therapies, removing several onerous requirements as well as loosening restrictions on which facilities can provide treatment.

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The Ongoing Challenges of Understanding Long COVID and Exploring Innovative Solutions

Perficient: Drug Development

In this comprehensive blog post, we will delve into the epidemiology, incidence, prevalence, costs associated with Long COVID, its impact on various stakeholders, traditional treatment approaches, and innovative solutions that leverage Artificial Intelligence (AI) to combat this ongoing challenge.

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The Uncertain Origins of Aspirin

Codon

Salicylic acid is likely the active compound that blocks the enzyme cyclooxygenase , giving aspirin its efficacy, and was used as a treatment for fever and gout before aspirin was developed. He treated fifty people over five years, all of whom he said were either cured or helped by the treatment. 4 A reproduction of the Ebers Papyrus.

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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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