Three will hunt for mutations underlying rare inherited diseases caused by a glitch in a single gene. (sciencemag.org)
Niemann - Pick is a group of rare inherited diseases in which patients can not metabolize cholesterol and other lipids properly within the cell. (sciencemag.org)
A relatively rare inherited disease in which the nerves that control movement mysteriously degenerate and disappear, it is the childhood equivalent of the better - known (but no better understood) amyotrophic lateral sclerosis. (discovermagazine.com)