Despite past failures, Denali's extensive pipeline and solid financial runway make it a compelling buy at current low stock ...
Interest in lysosomal storage diseases (LSDs) has increased significantly, because several new therapeutics received FDA approval in 2003. Though long overlooked by most major pharmaceutical ...
It’s been just six months since Zevra Therapeutics scored an FD | Niemann-Pick disease type C therapy Miplyffa became ...
The Metabolic Disease Laboratory provides services for the diagnosis of lysosomal storage diseases. The laboratory provides screening of urine for glycosaminoglycans, oligosaccharides, sialic acid, ...
Dr Anup Rawool, Consultant in Clinical and Cancer Genetics at Sahyadri Hospitals, Pune, discusses innovative therapies and ...
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Inability of cells to recycle fats can spell disease: Study reveals how a protein helps transport fats out of lysosomesIn a previous study, the NUS Medicine team has shown that if Spns1 does not work properly, it leads to a buildup of lipid waste inside cells, causing diseases known as lysosomal storage diseases ...
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 ...
Both of Genzyme's products are indicated for the treatment of Gaucher's disease, a rare lysosomal storage disorder resulting from a hereditary deficiency in the glucocerebrosidase enzyme.
Denali Therapeutics Inc.’s DNLI share price has dipped by 10.43%, which has investors questioning if this is right time to buy.
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