Researchers continue to hone in on how best to prevent blood clots, which cause strokes and heart attacks, without unintended side effects.

Preclinical study identifies possible therapeutic target for cardiovascular risk factors that avoid the common problem of an increased risk of fatty liver disease.

Percentage of all child health spending that went to orphan drugs rose 65% in five years, while families’ portion rose even faster.

The technique could help researchers find drugs that improve health and lengthen life.

Positive clinical trial points to possible additional option for patients with Lou Gehrig’s disease. A study investigator explains the excitement and the caution.

While the number of drugs for rare diseases affecting children has grown, nearly 7,000 rare diseases still lack treatment options.