Search Results - rare+diseases

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Antisense Oligonucleotides to Prevent Genetic Mis-Splicing for ALS/FTD Therapeutics
SHORT DESCRIPTION Antisense oligonucleotides (ASOs) that correct TDP‑43–dependent mis‑splicing of KCNQ2 thereby reducing neuronal hyperexcitability in ALS/FTD and related TDP‑43 proteinopathies. INVENTORS Evangelos Kiskinis* Northwestern University Feinberg School of Medicine, Department of Neurology Jonathan...
Published: 5/12/2026   |   Updated: 5/7/2026   |   Inventor(s):  
Keywords(s): ALS - Amyotrophic Lateral Sclerosis, FTD - Frontotemporal Dementia, Neurodegenerative disease, Neurologic disease, Neurology, Nucleic Acids, Rare diseases, Targeted therapy, Therapeutics
Category(s): Life Sciences > Therapeutics
Once-Weekly Glucocorticoid Steroid Dosing for Enhanced Muscle Function and Metabolic Health
SHORT DESCRIPTION A glucocorticoid regimen that preserves the benefits of steroids on muscle mass and function while mitigating many of the metabolic and growth‑related toxicities seen with daily dosing in various inflammatory, autoimmune, respiratory, and neuromuscular disease settings. INVENTORS Elizabeth McNally*, MD, PhD Northwestern...
Published: 5/11/2026   |   Updated: 5/7/2026   |   Inventor(s):  
Keywords(s): Aging, DMD - Duchenne muscular dystrophy, Immunology, Inflammation, Metabolic disorders, Muscle metabolism, Rare diseases, Small molecule, Therapeutics
Category(s): Life Sciences > Therapeutics
Mitigating aberrant Cdk5 Activation in SMA – A Novel Therapeutic Strategy Targeting Mitochondrial Function
SHORT DESCRIPTION A targeted strategy that mitigates aberrant Cdk5 activity to restore mitochondrial function and reduce motor neuron degeneration in SMA models. INVENTORS Yongchao Ma* Northwestern University Feinberg School of Medicine, Department of Pediatrics * Principal Investigator NU Tech...
Published: 5/7/2026   |   Updated: 4/27/2026   |   Inventor(s):  
Keywords(s): Adjunct therapy, Neurodegenerative disease, Neurologic disease, Neurology, Rare diseases, SMA - Spinal muscular atrophy, Targeted therapy, Therapeutics
Category(s): Life Sciences > Therapeutics
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: 5/7/2026   |   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
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 Division) Alexis...
Published: 4/30/2026   |   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
A peptidomimetic for LIMP-2 based activation of Glucocerebrosidase as a Gaucher/Parkinson’s Disease therapeutic
NU 2016-019 INVENTORS Dimitri Krainc* Michael Schwake SHORT DESCRIPTION LIMP-2-based peptidomimetic compounds to facilitate glucocerebrosidase activation in Gaucher and Parkinson’s Disease BACKGROUND Mutations in glucocerebrosidase (GCase) cause Gaucher Disease. Additionally, GCase mutations are a major risk factor in Parkinson’s Disease,...
Published: 5/6/2026   |   Updated: 6/2/2023   |   Inventor(s):  
Keywords(s): Neurodegenerative disease, Neurologic disease, PD - Parkinson's Disease, Rare diseases, Therapeutics
Category(s): Life Sciences > Therapeutics
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: 5/6/2026   |   Updated: 5/23/2022   |   Inventor(s):  
Keywords(s): AD - Alzheimer’s Disease, ALS - Amyotrophic Lateral Sclerosis, HD - Huntington's Disease, Neurodegenerative disease, Neurology, PD - Parkinson's Disease, Rare diseases, Small molecule, Therapeutics
Category(s): Life Sciences > Therapeutics
Nano-VEGF as a Treatment for Spinocerebellar Ataxia and Other Neurological Disorders
NU 2018-085 Inventors Punett Opal Samuel I. Stupp* Short Description A novel cost-effective neuropeptide with VEGF activity, less immunogenicity and great stability. Background Beyond its canonical role as an angiogenic factor, vascular endothelial growth factor (VEGF) has recently been implicated in several neurodegenerative diseases such...
Published: 5/12/2026   |   Updated: 1/15/2019   |   Inventor(s):  
Keywords(s): Biomaterials, Nanomaterials, Nanotechnology, Neurodegenerative disease, Neurologic disease, Neurology, Rare diseases, Therapeutics
Category(s): Life Sciences > Therapeutics