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

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 ...

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

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

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

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

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

Heidi Rehm explores steps to harness quality genomic data to support disease detection and the importance of collaboration in ...

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 "degenerative muscle disease" turns fish flesh into freezer-burnt-looking tissue that people shouldn't eat, the Missouri ...

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

On Friday, Notre Dame held its fourth Rare Disease Patient Advocacy Summit where speakers shared their experiences and ...