An Illinois family pushed for both boys to receive the potentially life changing medical treatment after one was approved and ...

A mysterious genetic disease wiped out generations of one family before science uncovered its cause. This talk reveals how a ...

You don't have to manage your health alone, even if you have a rare disease. Although 1 in 10 people in the U.S. live with a rare disease and 95% of all rare diseases do not have an FDA-approved ...

Lozano is a rare disease mom, neuroscience Ph.D. candidate at UC Davis, and board member for the PURA Syndrome Foundation. In May, a historic moment in science and medicine was captured in a single ...

The baby saved from a rare disease by a first-ever personalized gene fix has reached a big milestone, taking his first steps ahead of Christmas. KJ Muldoon is walking and getting ready to celebrate ...

President Lee Jae Myung (L), alongside his wife, Kim Hea Kyung, speaks during a meeting with patients stricken with rare diseases and their families at the heavy ion therapy center of Severance ...

U.S. health officials have added two deadly rare diseases to the list of conditions the government recommends states screen ...

For Americans living with rare diseases, the Rare Pediatric Disease Priority Review Voucher Program is a vital tool. Yet its reauthorization is at risk.

Uncommon illnesses caused by bacteria, viruses, fungi, or parasites fall under the category of rare infectious diseases. These illnesses are often caused by unique pathogens that affect very few ...

VHL, a genetic cancer condition, recently was featured on "Grey's Anatomy." Feb. 10, 2011 — -- In a multi-episode arc on "Grey's Anatomy," actor Scott Foley plays a von Hippel-Lindau (VHL) ...

When Casey McPherson became a father, his purpose became clear. He needed to raise his daughter, Rose, to be loving, courageous, healthy and strong. He needed to protect her. Many parents can ...

Rare diseases are defined as conditions affecting fewer than 200,000 people in the US or less than 1 in 2,000 in Europe. 1-2 While each disease is individually rare, collectively, they represent a ...