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FDA Panel Backs Full Approval of Paxlovid for COVID-19

An FDA panel recommended the agency grant full approval to nirmatrelvir-ritonavir (Paxlovid) for treating high-risk COVID-19. By a vote of 16-1 on Thursday, the Antimicrobial Drugs Advisory Committee said the totality of evidence supports the traditional approval of the oral antiviral, which has been widely used since late 2021 under an emergency use authorization to […]

Families Push Research Forward in Rare Diseases

This story was supported by a grant from the National Press Foundation in partnership with Fondation de France. For the main story on how mRNA is being investigated for its potential in rare diseases, click here. Kathy Stagni first met Charles Venditti, MD, PhD, at a meeting of her organization, the Organic Acidemia Association, more […]

COVID Vaccines Made mRNA a Household Name. How Can It Help in Rare Diseases?

This story was supported by a grant from the National Press Foundation in partnership with Fondation de France. For MedPage Today‘s related story on how families support rare disease research, click here. One of the hardest things about managing Jordan Franks’ propionic acidemia (PA) was feeding him. Jordan never wanted to eat. His body seemed […]

FDA Panel Unanimously Backs Moderna’s COVID Vax for Kids and Teens

A committee of independent vaccine experts recommended that the FDA grant an emergency use authorization (EUA) for the two-dose Moderna COVID-19 vaccine for kids ages 6 to 17 years. The Vaccines and Related Biological Products Advisory Committee (VRBPAC) voted 22-0, agreeing unanimously that the benefits of vaccination outweigh the risks in two age groups: kids […]

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