Prevalence of lysosomal storage disorders

PJ Meikle, JJ Hopwood, AE Clague, WF Carey - Jama, 1999 - jamanetwork.com
ContextLysosomal storage disorders represent a group of at least 41 genetically distinct,
biochemically related, inherited diseases. Individually, these disorders are considered rare,
although high prevalence values have been reported in some populations. These disorders
are devastating for individuals and their families and result in considerable use of resources
from health care systems; however, the magnitude of the problem is not well defined. To
date, no comprehensive study has been performed on the prevalence of these disorders as …