Search Results - rare+diseases

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JNK Inhibitors Targeting Bergmann Glia Inflammation to Mitigate Spinocerebellar Ataxia and Other Neurodegenerative Disorders
NU2021-060 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 INVENTORS Puneet Opal* Northwestern University Feinberg School of Medicine, Professor of Neurology Chandrakanth...
Published: 4/14/2026   |   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