There are more than 70 known lysosomal storage disorders (LSDs). These rare neurodegenerative diseases are characterized by the dysfunction of lysosomes, cellular organelles that break down ...

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 ...

In collaboration with a Stanford University research team, Scenic Biotech identified and characterized PLA2G15 as a novel target in lysosomal storage diseases ...

Individuals with the lysosomal storage disorder Niemann–Pick type C1 (NPC1) carry a mutation in the gene that encodes a protein called NPC1. Cells from people with the disease have defects in ...

Lysosomal Storage Disorders present significant challenges, including delayed diagnoses, fragmented care pathways, and limited access to specialist care. Digital health technologies (DHTs ...

Tenvie Therapeutics Inc. has described endosomal/lysosomal proton channel TMEM175 modulators reported to be useful for the treatment of cancer, neurological disorders, lysosomal storage disorders and ...

and Alzheimer’s disease. Gain has multiple undisclosed preclinical assets targeting lysosomal storage disorders, metabolic diseases, and solid tumors. Gain’s unique approach enables the ...

Gain Therapeutics, Inc. (Nasdaq: GANX) (“Gain”, or the “Company”), a clinical-stage biotechnology company leading the discovery and development of the next generation of allosteric small molecule ...

In collaboration with a Stanford University research team, Scenic Biotech identified and characterized PLA2G15 as a novel target in lysosomal storage diseases Amsterdam, the Netherlands ...

In collaboration with a Stanford University research team, Scenic Biotech identified and characterized PLA2G15 as a novel target in lysosomal storage diseases “BMP plays an integral role in promoting ...