Katherine Stueland, GeneDx CEO, joins 'Squawk Box' to discuss diagnosing children with rare diseases, cost structure of the ...

From symptoms to the illness' risk factors — here’s what you need to know about the disease, which can only be detected by ...

The NF-κB signaling pathway plays a pivotal role in how our bodies fight infection, heal, and regulate inflammation. At the ...

Regulators block Duchenne muscular dystrophy treatment after fatal side-effects outweigh questionable efficacy ...

Experts say the system of EDRDs incentivizes companies to manufacture these drugs due to the high prices that can be charged for them.

The pharmaceutical industry has a fresh sense of direction after President Donald Trump’s tax-and-spending law included a ...

It is trying to treat a devastating lifelong disease by fixing it at its source. We are able to fix the actual genetic ...

C3 glomerulopathy (C3G) and immune-complex membranoproliferative glomerulonephritis (IC-MPGN) are two diseases that have a ...

Rising high school senior Khartik Uppalapati says he felt alienated when he was diagnosed with a rare lymphatic disorder ...

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

MrBeast’s new CEO is hitting Wall Street as YouTuber Jimmy Donaldson’s media empire looks to develop long-term brand ...

Former Top Chef winner Hosea Rosenberg shared an update on his daughter Sophie’s journey with an extremely rare and painful ...