CME  Challenges of Diagnosing and Caring for an Individual with Gaucher Disease
The target audience for this CME Course is physicians and other providers, including nurse practitioners and physician assistants, who may encounter patients in their practice with undiagnosed Gaucher Disease… more info


Michael E. Shy, MD: Natural History, Biomarkers, and Gene Identification
Michael E. Shy, MD, Director, Division of Neuromuscular Medicine, Neurology at the University of Iowa Health Care, discusses Natural History, Biomarkers, and Gene Identification during the Rare Disease Day at NIH 2019 on Thursday, February 28, 2019 . . .  more info


Heather A. Lau, MD: Overview of the Mucopolysaccharidoses

Heather A. Lau, MD, Director, Lysosomal Storage Disease Program at NYU Langone Health provides an overview of the mucopolysaccharidoses (MPSs) a group of rare, inherited lysosomal storage disorders that are clinically characterized by abnormalities in multiple organ systems and reduced life expectancy. . .  more info