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Combination Treatment to Reduce Pathological Proteins in Neurodegenerative and Lysosomal Storage Diseases
NU 2021-206 INVENTORS Joseph Mazzulli* Iva Stojkovska SHORT DESCRIPTION A small molecule combination treatment to improve lysosomal function in neurodegenerative and lysosomal storage diseases BACKGROUND Many neurodegenerative disorders are characterized by a collapse in proteostasis, as shown by the accumulation of insoluble protein aggregates...
Published: 12/16/2025   |   Inventor(s):  
Keywords(s): AD - Alzheimer’s Disease, ALS - Amyotrophic Lateral Sclerosis, HD - Huntington's Disease, Neurodegenerative disease, Neurology, PD - Parkinson's Disease, Small molecule, Therapeutics
Category(s): Life Sciences > Therapeutics