A cold triggered an autoimmune disease in a Pa. man. Now he’s channeling his challenges into advocacy for people with rare diseases.

Brian Dawson's life changed when he received a misdiagnosis of neuromyelitis optica (NMO). As a patient advocate, he's now ...
STAT

Parents of children with rare diseases ask: How long until our CRISPR miracle?

In May, a historic moment in science and medicine was captured in a single photo that circulated across news outlets ...
KERA News

Texas is screening for more rare diseases in babies, but families still face barriers to treatment

Since the 1960s, Texas has screened newborns for a variety of rare diseases — like sickle cell anemia and cystic fibrosis.

Trial results show belzutifan shrinks rare neuroendocrine tumors and improves symptoms in patients

A multicenter Phase II clinical trial led by researchers at The University of Texas MD Anderson Cancer Center demonstrated ...

Contributor: By loosening standards, the FDA isn't doing rare-disease patients any favors

It's reasonable to assess rare-disease drugs differently. That's why the FDA has been remarkably flexible about these ...

Thousands of People Attended a Parade to Cheer Up a Terminally Ill 3-Year-Old Boy with a Disease Known as 'Butterfly Skin'

Thousands of people attended a parade to help cheer up a boy from Kansas City, Mo., who suffers from a rare genetic disease ...
USA Today

Her 5-year-old has a severe, rare disease. There's a cure, but she can't get it.

Catherine Illingworth, 39, knew something was wrong the moment her son's neurologist walked into the room. Illingworth requested the referral from her son's pediatrician based on instinct. George took ...
Fierce Biotech

'Some encouraging signs in the market': Electra shines with $183M fundraise for rare disease trial

Electra Therapeutics is incandescent, glowing on the heels of a $183 million series C financing intended to fuel the ...

Alan Jackson, 67, Opens up About His CMT Symptoms: ‘It’s Getting More Obvious'

When legendary country singer Alan Jackson, 67, announced that the “finale” show of his farewell tour next year will support ...

What is Kawasaki disease? How to recognize the symptoms in your child

Kawasaki disease is most likely to affect children under five years old and of Asian heritage. Here's how to recognize the ...
ABC News

'Grey's Anatomy' Features Rare Disease on Three-Episode Series

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) ...