"Squawk Box" co-host Becky Quick launched CNBC Cures in January, the network's initiative to raise awareness for rare ...

Life-saving treatments for children with orphan illnesses often move slowly, if at all, through the federal approval process.

Stanford Medicine pediatric hematologist Agnieszka Czechowicz, MD, Ph.D., has devoted her research career to improving ...

Prenatal genetic testing has existed since the 1970s. In the 1990s, non-invasive testing became available, and significant scientific gains were made. Now, doctors can screen for hundreds of genetic ...

This article was featured in New York’s One Great Story newsletter. Sign up here. The first nine days of Jorie Kraus’s life followed a pattern: problem, solution. Problem, solution. Problem: Jorie was ...

When Meenu Hodiwalla gave birth to her son almost three decades ago, she imagined the early days unfolding like they do for most new parents - tiny fingers curling around hers, the baby's eyes ...

A Beaverton family that has spent years searching for a replacement gene for their son’s rare genetic disorder says they are ...

The FDA has outlined a new review process for drugs and biologics designed to treat ultrarare genetic diseases that would allow a single-arm trial, plus other supportive data, to serve as pivotal ...

Connecticut prides itself on strong schools, world-class healthcare, and a commitment to caring for its most vulnerable residents. We are a state that values education, innovation, and community. Yet ...