The firm sees the astonishingly high cost of "cellular, gene and RNA therapies" — high-tech treatments for conditions like ...

In head-to-head clinical trials, ALYFTREK was non-inferior on ppFEV1 and superior in reducing sweat chloride compared to ...

Acute chest syndrome associated with sickle cell is driven by the abnormal activation of the complement cascade, per a study.

What if a heart attack and lifetime of statin drugs could be avoided with a one-time, two-hour infusion? Kiran Musunuru, a University of Pennsylvania cardiologist and leading gene-editing researcher, ...

Insiders have become bearish. In fact, according to MarketWatch, “Corporate insiders have taken a sharply pessimistic turn – ...

Vertex Pharmaceuticals is all set to release its fiscal second-quarter earnings next month, and analysts project a robust ...

PRNewswire/ - Medunik USA, a specialty pharmaceutical company dedicated to providing rare disease patients with access to ...

Sickle cell disease treatment progress has improved over the years, but recent federal cuts threaten the health of those who suffer from the blood ailment.

Sofia, 11, wouldn't be enjoying the simple pleasures of swimming, riding a bike and spending time with friends today had it ...

In a country where 90 per cent of children with sickle cell disease once died before their fifth birthday, Betty, 64, and ...