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Pyrimidine-2,4,6-Trione Derivatives Preparation and Their Use for the Treatment of Amyotrophic Lateral Sclerosis (ALS) ALS Therapy
NU 2009-072
INVENTORS
Richard Morimoto*
Richard Silverman*
Gavin Guoyao Xia
SHORT DESCRIPTION
This invention discloses novel pyrimidine-2,4,6-trione derivatives (PYT compounds) that prevent abnormal protein aggregation associated with ALS, offering a promising new therapeutic approach for neurodegenerative diseases.
BACKGROUND
Amyotrophic...
Published: 11/18/2025
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Inventor(s):
Keywords(s): ALS - Amyotrophic Lateral Sclerosis, Neurodegenerative disease, Therapeutics
Category(s): Life Sciences > Therapeutics
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Therapeutic Intervention for ALS and Dementia
NU 2021-058
INVENTORS
Evangelos Kiskinis*
Juan Alberto Ortega Cano Marco Boccitto
SHORT DESCRIPTION
Design of an RNA-based oligonucleotide that protects from dipeptide repeat protein toxicity for the treatment of ALS and FTD.
BACKGROUND
Amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD) are devastating neurodegenerative diseases...
Published: 12/18/2025
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Inventor(s):
Keywords(s): ALS - Amyotrophic Lateral Sclerosis, CNS - Central Nervous System, DPR - Dipeptide Repeat Proteins, FTD - Frontotemporal Dementia, Neurodegenerative disease, Neurology, RNA - Ribonucleic Acid, Targeted therapy, Therapeutics
Category(s): Life Sciences > Therapeutics
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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
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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
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