A mother has spoken about her "painful" experience over the potential end to her son's life-enhancing treatment for a rare ...

Last year, a series of essays profiling quintessential public servants made up our “Who Is Government?” project. The effort ...

It’s been just six months since Zevra Therapeutics scored an FD | Niemann-Pick disease type C therapy Miplyffa became ...

The estate of late actor Gene Hackman is seeking to block the public release of autopsy and investigative reports related to ...

Company expects to submit New Drug Application (NDA) in April 2025 - DEER PARK, Ill., March 14, 2025 (GLOBE NEWSWIRE) -- Eton ...

With thousands of rare diseases affecting millions of families worldwide, one Tampa Bay lawmaker is taking action to position Florida as a national leader in genetic medicine.

Data on the number of cases for these diseases from the pre-vaccine era is hard to come by, and even the case counts are ...

Jaguar expects first results in Q2 2025 of proof-of-concept investigator-initiated trials of crofelemer for the rare diseases short bowel syndrome with intestinal failure and microvillus inclusion dis ...

STATEN ISLAND, N.Y. — A fundraising Polar Plunge to honor John Hudson Dilgen will take place this month in South Beach to ...

A seasoned cattle station worker was only a few weeks into his first stint on a Far North Queensland property when he ...

The estate is seeking to block the public release of autopsy and investigative reports, especially photographs and video.

As health care costs continue to rise and the burden of chronic disease grows, data-driven insights will be essential in ...