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.
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.