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 ...

ANI Pharmaceuticals Targets $1B+ 2026 Revenue as Rare Disease Pivot Accelerates, Cortrophin Leads Growth

ANI Pharmaceuticals (NASDAQ:ANIP) outlined plans to accelerate its shift toward rare disease medicines while maintaining profitability and cash generation from its generics business, according to ...
BioSpace

Sentynl, Fortress Bounce Back With FDA Approval for Rare Pediatric Disease

The FDA previously rejected Zycubo for Menkes disease in October last year, citing issues with the drug’s manufacturing ...
Becker's Hospital Review

FDA approves 1st treatment for rare pediatric disease

FDA approves 1st treatment for Menkes disease, offering a copper therapy that improves survival in affected children.
Opinion
Dayton Daily NewsOpinion

Opinion: Protect access to life-saving treatments for Ohioans with rare diseases

As someone living with a rare disease in Ohio, I know how fragile access to life-saving treatment can be. A bill to help patients with rare or life-threatening illnesses is currently being debated in ...