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Research reveals therapeutic potential of mRNA in rare diseases

Drug Discovery World

Researchers have used messenger RNA (mRNA) to create an effective therapy for a rare liver disease in preclinical studies, demonstrating the technology’s potential therapeutic use in people. Patients affected by the disease are found to also experience an imbalance of glutathione regulation, which is important for liver detoxification.

Disease 148
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Preliminary Findings from a Phase 2a Trial of Investigational COVID-19 Therapeutic Molnupiravir

The Pharma Data

The companies today reported findings on one secondary objective from the Phase 2a study, showing a reduction in time (days) to negativity of infectious virus isolation in nasopharyngeal swabs from participants with symptomatic SARS-CoV-2 infection, as determined by isolation in Vero cell line culture. This press release features multimedia.

Trials 69
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Early Data Suggest Pfizer Pill May Prevent Severe COVID-19

NIH Director's Blog: Drug Development

The first indication that those hopes might be realized came from the announcement just a month ago of a 50 percent reduction in hospitalizations from the Merck and Ridgeback drug molnupiravir (originally developed with an NIH grant to Emory University, Atlanta). But would it work to treat COVID-19 in an infected person?

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Veklury®(Remdesivir) Efficacy and Safety Profile Further Demonstrated in Vulnerable Patient Populations

The Pharma Data

A separate real-world analysis demonstrated that use of Veklury is also associated with reduced hospital readmission risk in immunocompromised patients hospitalized with COVID-19. Since the beginning of the pandemic, Veklury has played a critical role in the treatment of hospitalized patients with COVID-19. 0.75; p<0.0001).

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Phase 2/3 Studies of Molnupiravir, an Investigational Oral Antiviral Therapeutic for Mild to Moderate COVID-19.

The Pharma Data

Merck (NYSE: MRK), known as MSD outside the United States and Canada, and Ridgeback Biotherapeutics announced today the presentation of previously announced Phase 2 interim results from two Phase 2/3 clinical trials (MOVe-OUT and MOVe-IN) of molnupiravir (MK-4482/EIDD-2801), an investigational oral antiviral therapeutic. About Molnupiravir.

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Merck and Ridgeback Biotherapeutics Announce Initiation of Pivotal Phase 3.

The Pharma Data

Merck (NYSE: MRK), known as MSD outside the United States and Canada, and Ridgeback Biotherapeutics announced today the initiation of the Phase 3 MOVe-AHEAD clinical trial to evaluate molnupiravir, an investigational oral antiviral therapeutic, for the prevention of COVID-19 infection.

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Eclectic Genomics: Cat Flu, Dolphin Adaptation to Climate Change, Predicting Cancer, and Diagnosing Rare Disease

PLOS: DNA Science

The owners of the felines all reported feeding their pets raw meat, and samples of the meat revealed not only flu virus genetic material, but also infectious virus. The fear is that a variant of the deadly virus might emerge that infects people, and, worst-case-scenario, passes from person to person. emerged in late 2021.

Disease 69