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Search Results - rare+diseases

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JNK Inhibitors Targeting Bergmann Glia Inflammation to Mitigate Spinocerebellar Ataxia and Other Neurodegenerative Disorders
SHORT DESCRIPTION Novel therapeutic strategy targeting Bergmann glia inflammation using JNK inhibition for treatment of spinocerebellar ataxia type 1 (SCA1) and other neurodegenerative disorders. INVENTORS Puneet Opal* Northwestern University Feinberg School of Medicine, Professor of Neurology Chandrakanth...
Published: 4/16/2026   |   Updated: 4/14/2026   |   Inventor(s):  
Keywords(s): Bergmann glia inflammation, Neurodegenerative disease, Neuroinflammation, Neurologic disease, Neurology, Rare diseases, SCA - Spinocerebellar ataxia, Small molecule, Therapeutics
Category(s): Life Sciences > Therapeutics
Recombinant Annexin Therapy for Muscle, Cardiac, and Neuronal Cell Membrane Repair
NU 2022-008 SHORT DESCRIPTION An annexin-based biological therapeutic that enhances the body's natural cell membrane repair process to reduce skeletal muscle, cardiac, and neuronal cell death and preserve tissue function. INVENTORS INVENTORS Elizabeth McNally* Feinberg School of Medicine, Department of Medicine (Cardiology...
Published: 4/14/2026   |   Updated: 4/14/2026   |   Inventor(s):  
Keywords(s): Biologic, Cardiomyopathy, DMD - Duchenne muscular dystrophy, Inflammation, Membrane injury and repair, Neurodegenerative disease, Rare diseases, SCI - Spinal cord injury, TBI - Traumatic brain injury, Therapeutics
Category(s): Life Sciences > Therapeutics
ASOs targeted towards mis-spliced UNC13A
NU2022-200 INVENTORS Wanhao Chi Jonathan Watts Evangelos Kiskinis* BACKGROUND Amyotrophic Lateral Sclerosis (ALS) is a progressive neurodegenerative that leads to 1 in 500 deaths in adults. There is no cure to stop or reverse ALS, thus there is a need to better understand its pathobiology and develop effective therapeutics. Northwestern...
Published: 4/13/2026   |   Updated: 10/17/2024   |   Inventor(s):  
Keywords(s): ALS - Amyotrophic Lateral Sclerosis, FTD - Frontotemporal Dementia, Neurodegenerative disease, Neurology, Nucleic Acids, Rare diseases, Targeted therapy, Therapeutics
Category(s): Life Sciences > Therapeutics