Genes are short sections of DNA that determine an animal’s traits—everything from coat color and claw strength to body shape ...

"Gene editing takes centre stage in FDA’s new rare disease approval pathway" was originally created and published by ...

Scientists at Gladstone Institutes have flipped the traditional approach to finding potential treatments for deadly diseases.

The Food and Drug Administration aims to evaluate treatments for rare diseases based on plausible evidence that they would work — without requiring a clinical trial first.

In many countries, newborn screening programmes test babies for certain inherited conditions shortly after birth.

The addition of PacBio to the iHope network will further expand diagnostic access worldwide DAMASCUS, MD, UNITED ...

St. Louis Families Unite for Rare Disease Day 2026 as Take Part Foundation Advances Critical Genetic Testing and ...

The plan could provide patients earlier access to treatments that would otherwise stall because only limited data can be gathered.

With 300 million people living with rare diseases, experts say new gene and cell therapies are giving families in Wisconsin hope for lasting treatment options.

During the Boston Globe’s Rare Disease Summit, Dr. Wendy Chung, a physician-scientist and geneticist who leads the Department ...