Sentences with phrase «of lysosomal storage»

In the lipid storage form of lysosomal storage disease I mentioned that dogs and cats can be born with decreased or missing lysosome enzymes that allows them to process fatty substances (lipids).

A type of lysosomal storage disease, this can cause skeletal abnormalities, growth retardation, and gait abnormalities, and can require close monitoring and special measu...

Lipidosis: a form of lysosomal storage disease where lipids accumulate in nerves.

An early onset form of lysosomal storage disease, this can cause affected dogs to display neurologic signs as puppies or young adults.

Not exact matches

The drug candidate is intended for the treatment of subjects with neuronal ceroid lipofuscinosis, aka Batten Disease, a fatal lysosomal storage disease that primarily affects the nervous system in children.

Genzyme has long been known for our expertise in the class of rare genetic diseases known as lysosomal storage disorders (LSDs).

Members of the section use the NIH Clinical Center to evaluate individuals with cystinosis, a lysosomal membrane defect leading to storage of the amino acid cystine and causing kidney failure in childhood.

Enhanced delivery of α - glucosidase for Pompe disease by ICAM -1-targeted nanocarriers: comparative performance of a strategy for three distinct lysosomal storage disorders.

It is one of over 50 genetically inherited disorders known as Lysosomal Storage Diseases.

GM1 gangliosidosis, or Landing disease, is a rare inherited neurodegenerative lysosomal storage disorder characterized by severe cognitive and motor developmental delays resulting in the death of most patients at a very young age.

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.

Both MPS IIIA and GM1 are classified as neuropathic (i.e. affecting the central nervous system) Lysosomal Storage Disorders, a group of approximately 50 rare inherited metabolic disorders that result from defects in lysosomal Lysosomal Storage Disorders, a group of approximately 50 rare inherited metabolic disorders that result from defects in lysosomal lysosomal function.