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.
In the past week, two announcements from the drug discovery world have had Insilico Medicine’s generative artificial intelligence (AI) target discovery engine, PandaOmics, front and center. The first ...
Credit: Getty Images. Laura Buch, MSPAS, PA-C, emphasizes the need for newborn screening to detect infantile-onset lysosomal storage diseases and swift treatment. In honor of PA Week 2024, we ...
3D rendering of a single live cell acquired through holographic tomographic flow cytometry (HTFC), showing the nucleus (purple) and aggregated lysosomes (green) within the cytoplasm (pink). This ...
The Metabolic Disease Laboratory provides services for the diagnosis of lysosomal storage diseases. The laboratory provides screening of urine for glycosaminoglycans, oligosaccharides, sialic acid, ...
For Zevra Therapeutics and its rare neurodegenerative disease med arimoclomol, the second time’s the charm. Friday, the FDA approved Zevra’s arimoclomol capsules to treat the rare lysosomal storage ...
For Zevra Therapeutics, last year’s approval of the rare lysosomal storage disorder drug Miplyffa appears to be the gift that keeps on giving. After receiving a rare pediatric disease priority review ...