I thought the hardest part of this journey would be to find a cure and a treatment for my son,” Andrew Jedlicka told The Post ...

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.

Harley, 15, was diagnosed with an extremely rare genetic disorder affecting his spine, hearing and vision. His mother, Kelly, ...

At an event on Monday, the agency officially unveiled long-awaited draft guidance meant to help speed the development of bespoke treatments for extremely rare diseases.

A new study has shed light on why patients with certain rare immune disorders develop severe, food-triggered allergic reactions while others with similar diagnoses do not. The findings, published in ...

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

An Ice Age double burial in Italy has yielded a stunning genetic revelation. DNA from a mother and daughter who lived over 12,000 years ago shows that the younger had a rare inherited growth disorder, ...

Growth charts for children with rare genetic disorders - giving healthcare professionals and families clearer guidance on how a child is developing - have been created by an international team, led by ...

KSHB 41 reporter Olivia Acree covers portions of Johnson County, Kansas. A viewer reached out to KSHB about this story, so Olivia sat down with the family. Share your story idea with Olivia. We are ...

KUALA LUMPUR - A recent viral story on social media tells of a fair-skinned baby with long eyelashes who, at first glance, appears no different from any other infant. Few would have guessed that the ...

LOUISVILLE, Ky. (WAVE) - Carter Twigg held her son Owen for the last time on Sept. 29, 2023, after he went to sleep and didn’t wake up. Owen Twigg was born with a cleft lip and palate and complex ...

Advances in DNA sequencing have revealed something surprising—not everyone with the same genetic change develops the same ...