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 ...

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.

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

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

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 father of a nine-year-old girl with a rare disease likened to childhood dementia has called for health bosses to prolong ...

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 ...

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 ...

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