It is also for these reasons that Rare Disease Day, which takes place every February and is coordinated by EURORDIS, is so important. The #LightUpForRare 8 theme highlights the issues faced by ...
October 19 is Niemann-Pick Day, focused on raising awareness about this rare genetic disease affecting children's metabolism ...
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 ...
On Rare Disease Day, Clinigen has launched a campaign to try to encourage doctors and patients to explore how they can get early access to investigational therapies. The ‘What is Possible?’ ...
The Minnesota Rare Disease Advisory Council works to help those with rare diseases get screened, diagnosed and treated.
Emma Austin needs $172,000 therapy every two weeks to slow the progression of Batten disease, which has "no known survivors." ...
After extensive testing, Penny was diagnosed with Dravet syndrome, a serious, genetic form of epilepsy. “When she was 11 months old, we received the results that she had a mutation of the SCN1A gene, ...
FA is a progressive neuromuscular disease that causes loss of coordination and mobility. My parents got my test results the ...
Archie Williams, nine, struggles to breathe and can't walk very far - but you wouldn't know anything was wrong by looking at ...
I’m not joking when I say your life can change in the blink of an eye, because that was what happened to us,” his grieving ...
The "brain-eating amoeba" causes a fatal infection of the brain and its protective layers. Here is everything you need to ...
By Jula Inrig, M.D. Chief Medical Officer, Travere Therapeutics | When a specialized field of medicine has long been quiet, a ...
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