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 ...
By identifying the causes of a rare condition known as auto-brewery syndrome (ABS), scientists can perfect methods for ...
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 joins 'Squawk Box' to discuss the company's 2026 outlook, drug pipeline, the focus on rare ...
The U.S. Food and Drug Administration has extended its review for the expanded use of Travere Therapeutics’ drug to treat a ...
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 ...
Elizabeth Diamond's husband, Chris, died after contracting a rare disease from swimming in a waterfall during their 2016 ...
After years stuck in the “doldrums,” the biopharma sector is in a “very good place” heading into the new year, analysts told ...
The Priority Review Voucher (PRV) program is designed to incentivize rare disease drug development.
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