Patients with rare diseases are increasingly working to find and fund their own cures. But should they have to?

Explosion in the tick pop­u­lation draws attention to rare Lyme disease symptoms in children BY Erin Billups - National Health Reporter Nationwide UPDATED 7:35 PM ET Jun. 21, 2021 ...

Measles is highly contagious among those who are unvaccinated; outbreaks can have severe health consequences in children ...

Senators made the right decision for America's most vulnerable patients ― now, the House must follow suit. The ORPHAN Cures Act is a simple fix to a costly mistake ― and a necessary step to keep rare ...

R136S, or the “Christchurch mutation,” appears to protect against Alzheimer’s disease by suppressing inflammatory signaling ...

The designation reinforces the potential of QRX003 as a therapeutic candidate for a profoundly underserved pediatric population. It follows earlier regulatory recognition by the European Medicines ...

The FDA rejected Stealth BioTherapeutics’ latest bid to market its proposed treatment for an ultra-rare genetic condition, though regulators have suggested a “path” toward accelerated approval.

The term “rare disease” comprises more than 7,000 diseases, each affecting fewer than 200,000 Americans, collectively impacting 25 million to 30 million individuals. 1 Exome sequencing tests ...

The term “rare disease” comprises more than 7,000 diseases, each affecting fewer than 200,000 Americans, collectively impacting 25 million to 30 million individuals. 1 Exome sequencing tests – which ...

The NHS is “not working” for people in the UK with rare diseases as they are left to “fall through the cracks,” a damning new report has claimed. Nearly three in ten (30 per cent) people ...

Two people have died from a rare brain disorder similar to Mad Cow disease, according to the Hood River County Health Department. Health officials said three cases of Creutzfeldt-Jakob disease, or CJD ...

Acquisition of Theravia from Mérieux Equity Partners represents meaningful step forward in Norgine's strategy for sustainable growth, adding a complementary portfolio of rare disease medicines ...