The University of Minnesota’s annual Rare Disease Day seminar this year, held at the end of February, explained how patients with rare diseases can utilize telehealth services nationally and ...

As Rare Disease day 2020 approaches, we take a look at the biggest challenges facing orphan drug developers and ask whether the future is bright or bleak for these difficult conditions.

To mark upcoming Rare Disease Day, pharmaphorum asked a panel of experts to give their thoughts on the opportunities and challenges facing developers of rare disease therapies in 2024. The panel ...

FA is a progressive neuromuscular disease that causes loss of coordination and mobility. My parents got my test results the ...

October 19 is Niemann-Pick Day, focused on raising awareness about this rare genetic disease affecting children's metabolism ...

She feared for her life every day for three years. “It was extremely depressing,” said Suki. “Everyone wants to see their ...

The Minnesota Rare Disease Advisory Council works to help those with rare diseases get screened, diagnosed and treated.

By the time Noah Jordan was diagnosed with a rare mitochondrial disease, his parents were told there was no cure ...

“That's the problem with the rare disease community as a whole,” she explains ... “My goal, at the end of the day, is to get ...

Archie Williams, nine, struggles to breathe and can't walk very far - but you wouldn't know anything was wrong by looking at ...

Most boys treated with breakthrough gene therapy for a rare but deadly brain illness are faring well six years later, two new ...