Search Results - ehud+goldin

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Glucocerebrosidase Non-inhibitory Chaperones for the Treatment of Gaucher Disease, Parkinson's Disease, and Other Proteinopathies
Gaucher disease is a rare lysosomal storage disease that is characterized by a loss of function of the glucocerebrosidase (GCase) enzyme, which results in a decreased ability to degrade its lipid substrate, glucocerebroside. The intracellular build up of this lipid causes a broad range of clinical manifestations, ranging from enlarged spleen/liver...
Published: 10/28/2024   |   Inventor(s): Wei Zheng, Samarjit Patnaik, Noel Southall, Ellen Sidransky, Ehud Goldin, Wendy Westbroek, Steven Rogers, Frank Schoenen, Juan Marugan
Keywords(s): CHAPERONES, Gaucher Disease, GLUCOCEREBROSIDASE, IBXXXX, NB1BXX, NBXXXX, NON-INHIBITORY, Parkinson's Disease, proteinopathy, UAXXXX, VEXXXX, WKXXXX, YAXXXX, YBXXXX
Category(s): Collaboration Sought > Collaboration, Collaboration Sought > Licensing, Application > Therapeutics, TherapeuticArea > Neurology, TherapeuticArea > Rare / Neglected Diseases
Glucocerebrosidase Activators as a Treatment for Gaucher Disease
This technology is a collection of small molecule activators of a genetically defective version of the enzyme called glucocerebrosidase (GCase), which causes Gaucher disease. Gaucher disease is a rare disease affecting 1 in 40,000 babies born. Ashkenazi Jews of eastern European descent (about 1 in 800 live births) are at particular risk of carrying...
Published: 10/28/2024   |   Inventor(s): Noel Southall, Ehud Goldin, Wei Zheng, Samarjit Patnaik, Ellen Sidransky, Omid Motabar, Wendy Westbroek, Juan Marugan
Keywords(s): ACTIVATORS, Disease, Gaucher, GLUCOCEREBROSIDASE, IA1AXX, IA1XXX, IAXXXX, IB6XXX, IBXXXX, IXXXXX, treatment
Category(s): Collaboration Sought > Licensing, TherapeuticArea > Reproductive Health, Application > Research Materials, Application > Diagnostics, Application > Therapeutics, Application > Vaccines, TherapeuticArea > Cardiology