Harvey Weinstein, the Hollywood producer whose predatory behavior sparked the Me Too movement and landed him in prison, has ...

It was very scary.” They found out she had a rare condition called systemic mastocytosis, a condition that affects every ...

At #STATSummit, three parents discussed the challenges they've faced trying to find treatments for their children with rare ...

Fifty years ago, a memorial fund for research in Reye’s disease was being established at Albany Medical College.

Over 10,000 rare diseases are currently affecting more than 30 million Americans. This article, written in partnership with members of the Rare Disease Compliance Consortium, focuses on the role of ...

Savara Inc. (Nasdaq: SVRA) (the Company), a clinical stage biopharmaceutical company focused on rare respiratory diseases, ...

Pitts, a former FDA associate commissioner, is president of the Center for Medicine in the Public Interest and a visiting ...

Coming on the heels of an advisory committee in which the U.S. FDA and its independent advisers grappled with trying to fit ...

Emma Austin needs $172,000 therapy every two weeks to slow the progression of Batten disease, which has "no known survivors." ...

New research demonstrates the potential of precise genome editing technologies, called adenine base editors, to correct disease-causing mutations in stem cells from patients with X-linked chronic ...

The Texas-based biotech sponsored a recent episode of “The Balancing Act,” a morning talk show that airs on Lifetime. Per an ...

The tool, called RADIANT, is designed to gather data in real time from the electronic medical records of children with rare ...