This new analysis shows early diagnosis of some lysosomal storage disorders can help avoid irreversible damage across the board. Lysosomal storage disorders (LSDs), including Gaucher disease (GD), ...
December 2, 2011 — Lysosomal storage disorders such as Fabry's disease and Pompe's disease are much more common than previously thought, particularly atypical later-onset forms, a new study suggests.
Main takeaways: Scientists at the Icahn School of Medicine at Mount Sinai in New York, the National Center for Advancing Translational Sciences (NCATS), and elsewhere have reversed the effects of ...
Cholesteryl Ester Storage Disease (CESD) is a rare autosomal recessive multisystem condition that belongs to the lysosomal storage disorders family. The liver is one of the most common sites of ...
LONDON--(BUSINESS WIRE)--Technavio’s latest market research report on the global lysosomal storage diseases (LSDs) market provides an analysis of the most important trends expected to impact the ...
A Denali treatment for a rare lysosomal storage disorder, Hunter syndrome, has posted encouraging biomarker results in a small, open-label Phase 1/2 trial. The genetic disorder is caused by mutations ...
Lysosomes are critical for cellular degradation, characterized by their acidic pH and array of hydrolytic enzymes. They degrade materials through endocytosis, phagocytosis, and autophagy, recycling ...
The Metabolic Disease Laboratory provides services for the diagnosis of lysosomal storage diseases. The laboratory provides screening of urine for glycosaminoglycans, oligosaccharides, sialic acid, ...
The MarketWatch News Department was not involved in the creation of this content.-- Dr. Abribat has a proven track record of developing innovative treatments for rare diseases, ac ...
Gain Therapeutics, a preclinical biotech developing small molecule therapies for lysosomal storage disorders, raised $40 million by offering 3.6 million shares at $11, within the range of $10 to $12.