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.
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), ...
The Indian Medical Parliamentarians Forum has urged urgent government action to prevent life-threatening treatment ...
An analysis of dried blood spots from around 35 000 babies in Austria has shown that lysosomal storage disorders—those in which the lysosome or 'recycling centre' of the body's cells malfunctions—are ...
Please provide your email address to receive an email when new articles are posted on . An Italian pharmaceutical company and a Boston-area biotechnology research firm announced a partnership to ...
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 ...
Indian Medical Parliamentarians’ Forum urges urgent government action to prevent treatment disruptions for children with rare ...
ST. LOUIS--(BUSINESS WIRE)--M6P Therapeutics (“M6PT” or “the Company”), a privately held life sciences company developing next-generation enzyme replacement and gene therapies for lysosomal storage ...
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 ...
HYOGO, Japan--(BUSINESS WIRE)--JCR Pharmaceuticals Co., Ltd. (TSE: 4552) made significant contributions at the Society for the Study of Inborn Errors of Metabolism (SSIEM) Annual Symposium 2024, held ...
Dr. Abribat has a proven track record of developing innovative treatments for rare diseases, achieving three successful ...
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 ...