My Son Has a Rare Disease. This Simple Test Could Have Changed Everything

We existed in our own world that day, absorbing information we never wanted to learn. At one point, while I was crying ...
FOX6 News Milwaukee on MSN

Muskego family helps raise millions for rare childhood disease treatment

A Muskego family helped raise more than $5.5 million to fund early access treatment for children with Sanfilippo syndrome ...
MedPage Today on MSN

Rare Childhood Disorder Gets Its First FDA Drug Approval

The FDA approved copper histidinate (Zycubo) as the first treatment for Menkes disease in pediatric patients, the agency ...

Scientists Healed a Man’s Rare Gut Disorder By Giving Him Someone Else's Poop

By identifying the causes of a rare condition known as auto-brewery syndrome (ABS), scientists can perfect methods for ...

Travere shares fall as US FDA extends review of rare kidney disease drug

The U.S. Food and Drug Administration has extended its review for the expanded ​use of Travere Therapeutics’ drug to treat a ...

Biogen CEO Chris Viehbacher on 2026 drug pipeline outlook, rare disease strategy

Biogen CEO Chris Viehbacher joins 'Squawk Box' to discuss the company's 2026 outlook, drug pipeline, the focus on rare ...

US FDA extends review of Travere’s drug for rare kidney disease

The U.S. Food and Drug Administration has extended its review for the expanded use of Travere Therapeutics’ drug to treat a ...

JPM26: 2025 a “breakout year” for rare disease biotechs

Full pipelines and rising revenues lead rare disease biotechs to restyle themselves as more mature, global biopharma ...
MedPage Today on MSN

New CDC Guidance Could Revive a Rare but Deadly Disease

The new CDC recommendation takes a much narrower view of the meningococcal disease threat. Because of "the low incidence ...

Singer reveals rare disorder could prevent twin daughters from walking

"We were told that they're probably never going to be able to walk," the 34-year-old tearfully explained in the clip.

Woman Had the Wedding of Her Dreams. Then Her Husband Died from a Rare Disease He Contracted While Swimming on Honeymoon

Elizabeth Diamond's husband, Chris, died after contracting a rare disease from swimming in a waterfall during their 2016 ...

13-Year-Old Girl Reveals the Contents of Her Bucket List Ahead of Losing Her Vision Due to Rare Genetic Disorder

Audrey Tyrell, 13, was diagnosed with a rare genetic disorder called Usher syndrome at age 10. The teen has created a bucket ...