Maryland-based Vtesse will work with the US National Institutes of Health to advance treatments for Niemann-Pick Type C disease and other lysosomal storage disorders. A series of rodent experiments ...

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 ...

The STING protein, known for helping cells fight viral infections by generating inflammation, also appears to function as a ...

AI and digital health have tremendous potential to improve patient outcomes across healthcare, but in rare disease – where ...

The Metabolic Disease Laboratory provides services for the diagnosis of lysosomal storage diseases. The laboratory provides screening of urine for glycosaminoglycans, oligosaccharides, sialic acid, ...

Metachromatic leukodystrophy (MLD) is an autosomal recessive lysosomal storage disease originating from biallelic pathogenic variants in the ARSA gene, mainly affecting young children. ARSA encodes ...

Genes associated with lysosomal dysfunction increase the risk of Parkinson’s disease (PD), according to a study led by ...

SALT LAKE CITY — Denali Therapeutics is advancing the fight against neurodegenerative diseases thanks to pioneering ... neurodegenerative diseases and lysosomal storage diseases, in Salt Lake ...

Mucopolysaccharidoses (MPS) are lysosomal storage disorders that are rare but debilitating inherited diseases associated with enzyme deficiencies. They typically become evident in the first 12 months ...

Thiruvananthapuram: Health minister Veena George announced the launch of the Vishu Kaineettam project, aimed at supporting ...