The objective of the fellowship is to provide an in - depth clinical research training experience at a premier medical center with expertise and significant clinical volume in the area of biochemical genetics, including
lysosomal storage diseases,
as well
as in therapeutics and clinical trials involving patients with these and other metabolic
diseases and, thereby, to increase the number of medical geneticists with interest, knowledge, and expertise in this area.
Lysosomal storage diseases such
as Niemann - Pick
disease, mucopolysaccharidosis and gangliosidosis (GM1) have been described in Siamese cats,
as has systemic amyloidosis.