POLG-related diseases disrupt the function of the mitochondria, or "powerhouses" of the cell — starving them of energy.

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 ...

Cena Hermus, 7, was two years old when she was diagnosed with a rare, life-threatening disorder that causes severe obesity.

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

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 ...

Gene Hackman’s nephew has spoken about funeral plans for the late star. The two-time Oscar winning actor, 95, died of heart ...

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

Senators told the Trump administration that Dr. Dave Weldon didn’t have the votes to be confirmed, though he had believed his ...

Pupils at Bridport Primary School put the spotlight on rare diseases by dressing in a riot of colour and learning about the challenges faced by those affected. As part of Rare Disease Day, children at ...

Filmmaker Mei Fa Tan reflected on her experience working with Luxembourg’s Prince Frederik before his death at the age of 22.