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

Researchers have found that a novel drug combination disrupts multiple factors in an aggressive type of breast cancer.

A federal “right-to-try” law for medical treatments could soon become reality. But systems to expand access to investigational options while protecting patients are already in place, says a U-M expert.

Ketamine is used increasingly during surgery to reduce pain during recovery. A recent study questioned the efficacy of this common practice.

Tissue and blood samples taken from patients can hold the keys to biomedical discovery. But the research community must consider patient privacy, a new perspective says.

How a new therapy came to be, from discovery to something used by patients everywhere, for Gaucher disease and perhaps more.