Popular weight-loss drugs linked to rare 'pirate disease,' researchers warn

Australian researchers warn GLP-1 weight-loss drugs may cause scurvy, the historic "pirate disease," due to poor nutrition ...

US FDA declines to approve Disc Medicine's rare disease drug

Disc Medicine said on Friday the U.S. Food and Drug Administration has declined to approve its drug to treat a rare genetic disorder.
Fierce Biotech

FDA rejects Disc’s rare disease drug despite commissioner’s voucher

The FDA has rejected a rare blood disease candidate from Disc Medicine, a compound that been granted expedited review through ...
STAT

FDA rejects rare disease therapy from Disc Medicine, early recipient of commissioner’s voucher

The FDA has rejected a rare disease therapy from Disc Medicine, an early recipient of a commissioner's voucher from the ...

Norwegian Cruise Line ship may be linked to cases of rare disease

Norwegian Cruise Line is working with the CDC after two guests that had sailed on Norwegian Encore this past December were ...
Fierce Biotech

Biohaven CEO calls recent FDA decisions ‘a systemic problem’

After a string of high-profile regulatory rebukes, Biohaven’s CEO believes red tape is getting in the way of patient ...

Drug for rare genetic disorder goes from FDA priority to reject in four months

The rejection of Disc Medicine Inc.’s experimental compound for a rare and devastating disorder by the US Food and Drug Administration, just months after it received a voucher intended to speed US ...

Parents share how they are fighting a rare genetic disorder affecting 1 in 1 million kids

Leena Panwala and Tim Drury share their stories with "CBS Mornings" about how they're raising awareness and money for INAD, a rare genetic disorder. Panwala created the INAD Cure foundation after her ...

Tennis great opens up about living with rare condition

Monica Seles has spoken publicly about how living with myasthenia gravis has affected her daily life. In a recent interview, ...

Machesney Park woman pleads for better care after rare disorder leaves her blind

The Machesney Park resident estimates spending nearly $1000 per month on eye treatments to avoid irritation; one regimen ...

Ultragenyx to cut 130 jobs following failed rare-disease drug trial

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Man awarded $25M after getting lung disease from popular cooking spray: ‘Nothing will give him his health back’

A Los Angeles man was awarded $25 million after developing a rare chronic lung disease known as “popcorn lung” from an ingredient that was used to be in the popular cooking spray PAM. Last week, a ...