Rare Diseases

Common diseases get much of the attention, but rare diseases also have a serious impact on people’s lives—and disproportionately affect children. Some have unfamiliar names: Hashimoto’s encephalitis, cystinosis, Charcot-Marie-Tooth type 1A. Others are better known, often for their severity: hemophilia, ALS, Huntington’s disease. With rapid advances in genomics, cell biology, neuroscience and high-speed drug screening, we now have an unprecedented opportunity to bring relief to people with diseases that currently offer few if any medical options.