Search Results - clinical+and+disease+specializations+%3e+neurology+%3e+amyotrophic+lateral+sclerosis

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Method to Inhibit Toxic Pathways Activated in Genetic ALS/FTD

Value PropositionTreatment readily acts via subcutaneous injection and crosses the blood-brain barrier without extra formulation or drug delivery mechanism.Treatment significantly reduces pathological and behavioral phenotypes from toxic proteins coded by genetic defects in amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD), which...

Published: 3/14/2025 | Inventor(s): Shuying Sun, Zhe Zhang

Keywords(s):

Category(s): Clinical and Disease Specializations > Neurology, Clinical and Disease Specializations > Neurology > Amyotrophic Lateral Sclerosis, Clinical and Disease Specializations > Neurology > Neurodegeneration, Clinical and Disease Specializations > Neurology > Motor Neuron Diseases, Clinical and Disease Specializations > Neurology > Dementia, Technology Classifications > Therapeutic Modalities > Peptides, Technology Classifications > Therapeutic Modalities > Targets, Technology Classifications > Diagnostics > Biomarkers

Mitigation of Nuclear Pore defect in Neurodegeneration: ESCRT Pathway Inhibition

Value PropositionNovel Target: CHMP7, a nuclear pore protein, identified as a key driver in ALS, offers a new therapeutic approach.Disease-Modifying Method: Targets the root cause of nuclear pore complex injury and neural injury, aiming to alter disease progression.Druggable Target: CHMP7 can be inhibited using small molecule, gene, and peptide therapies.Broad...

Published: 3/14/2025 | Inventor(s): Jeffrey Rothstein, Alyssa Coyne

Keywords(s):

Category(s): Clinical and Disease Specializations > Neurology > Amyotrophic Lateral Sclerosis, Clinical and Disease Specializations > Neurology > Dementia, Clinical and Disease Specializations > Neurology > Alzheimer's Disease, Clinical and Disease Specializations > Neurology > Huntington's Disease, Clinical and Disease Specializations > Neurology > Neurodegeneration, Technology Classifications > Therapeutic Modalities > Targets

Methods to Treat Neurodegenerative Diseases

Unmet NeedAn estimated 930K people in the US are living with a neurodegenerative disease (Alzheimer’s and Parkinson’s are most common). However, current standard of care treatments mostly addresses symptoms of these diseases. To date no disease modifying or neuroprotective treatments are approved for Parkinson, and only 1 recently has been for Alzheimer’s...

Published: 3/14/2025 | Inventor(s): Rana Rais, Barbara Slusher, Arindom Pal

Keywords(s):

Category(s): Technology Classifications > Therapeutic Modalities, Technology Classifications > Therapeutic Modalities > Small Molecules, Clinical and Disease Specializations > Neurology > Alzheimer's Disease, Clinical and Disease Specializations > Neurology > Multiple Sclerosis, Clinical and Disease Specializations > Neurology > Amyotrophic Lateral Sclerosis, Clinical and Disease Specializations > Neurology > HIV-Associated Neurocognitive Disorders

Antisense oligonucleotide therapy and biomarker assay for C9ORF72 hexanucleotide expansion disorders

Therapy to Treat and Assay to Monitor Neurodegenerative Disease JHU REF: [C12195]Invention novelty: This technology is the use of antisense oligonucleotides (ASO) as a therapeutic for specific neurodegenerative diseases. The technology could also be used in development of a biomarker assay to monitor the efficacy of the ASO therapy. Value Proposition: Current...

Published: 3/13/2025 | Inventor(s): Christopher Donnelly, C. Bennett, Susan Freier, Rita Sattler, Jeffrey Rothstein

Keywords(s): Amyotrophic Lateral Sclerosis, Assay, Biologics, Clinical Diagnostics, CNS and Neurological Disorders, Dementia, Disease Indication, In Vitro Diagnostics, Neurodegeneration, Nucleic Acid, Therapeutic Matter, Therapeutic Substance, Therapeutics

Category(s): Clinical and Disease Specializations, Technology Classifications > Therapeutic Modalities > Biologics, Technology Classifications > Therapeutic Modalities > Gene Therapies, Technology Classifications > Diagnostics > In Vitro Diagnostics, Technology Classifications > Research Tools > Assays, Technology Classifications > Diagnostics, Clinical and Disease Specializations > Neurology, Technology Classifications > Research Tools, Technology Classifications > Therapeutic Modalities, Clinical and Disease Specializations > Neurology > Amyotrophic Lateral Sclerosis, Clinical and Disease Specializations > Neurology > Dementia, Clinical and Disease Specializations > Neurology > Neurodegeneration

Tonabersat as a Neuroprotective Compound for Amyotrophic Lateral Sclerosis

Unmet NeedAmytrophic Lateral Sclerosis (ALS) is a progressive neurodegenerative disease that affects motor neurons, and results in muscle atrophy and loss of motor control. ALS affects approximately 17,000 people in the United States and 450,000 worldwide at any given time, and the average life expectancy from diagnosis to mortality is between 2-5 years....

Published: 3/14/2025 | Inventor(s): Nicholas Maragakis

Keywords(s): Amyotrophic Lateral Sclerosis, Antagonists/Inhibitors, CNS and Neurological Disorders, Disease Indication, Non-novel, Predicted Novelty, Repurposed, Small Molecules, Therapeutic Matter, Therapeutic Substance, Therapeutics

Category(s): Technology Classifications > Therapeutic Modalities > Small Molecules, Technology Classifications > Therapeutic Modalities > Repurposed Drugs, Clinical and Disease Specializations > Neurology > Amyotrophic Lateral Sclerosis

Pharmacological Intervention of the Arachidonic Acid Pathway to Cure Amyotrophic Lateral Sclerosis

Unmet NeedAmyotrophic Lateral Sclerosis (ALS) affects as many as 30,000 people in the United States alone, with 5,000 new cases diagnosed every year. ALS is a fatal neuromuscular disease that is characterized by a progressive degeneration of the motor nerve cells and spinal cord. There are only two drugs approved by FDA for the treatment of ALS, and...

Published: 3/14/2025 | Inventor(s): Gabsang Lee, Hyungjin Eoh, Hojae Lee, Thomas Lloyd, Nicholas Maragakis

Keywords(s): Amyotrophic Lateral Sclerosis, CNS and Neurological Disorders, Disease Indication, Natural compounds, Non-novel, Predicted Novelty, Target, Therapeutic Matter, Therapeutics

Category(s): Clinical and Disease Specializations > Neurology > Amyotrophic Lateral Sclerosis, Technology Classifications > Therapeutic Modalities > Targets, Technology Classifications > Therapeutic Modalities > Repurposed Drugs

Stimulation of Dendrite Growth and Spine Formation by Norrin and LGR6: A Target for Therapy in CNS Injury and Degeneration

Unmet NeedAstroglial dysfunction is associated with the development of numerous central nervous system (CNS) diseases, including amyotrophic lateral sclerosis (ALS), Parkinson’s disease (PD), and Huntington’s disease (HD). Astroglia are the homeostatic cells of the central nervous system and play key roles in the development and support of neuronal...

Published: 3/14/2025 | Inventor(s): Sean Miller, Jeffrey Rothstein

Keywords(s): Alzheimer's Disease, Amyotrophic Lateral Sclerosis, Biologics, CNS and Neurological Disorders, Dementia, Disease Indication, Huntington's Disease, Parkinson's Disease, Protein, Target, Therapeutic Matter, Therapeutic Substance, Therapeutics

Category(s): Clinical and Disease Specializations > Neurology > Neurodegeneration, Clinical and Disease Specializations > Neurology > Amyotrophic Lateral Sclerosis, Clinical and Disease Specializations > Neurology > Dementia, Technology Classifications > Therapeutic Modalities > Proteins, Clinical and Disease Specializations > Neurology > Alzheimer's Disease, Technology Classifications > Therapeutic Modalities > Targets, Clinical and Disease Specializations > Neurology > Huntington's Disease, Clinical and Disease Specializations > Neurology > Parkinson’s Disease

Target Validation of Splicing Repression, a Major Function of TDP-43 in the Motor Neuron

Unmet NeedAmyotrophic lateral sclerosis (ALS) is a rare but debilitating disease characterized by the death of motor neurons that eventually leads to difficulty moving, speaking, swallowing, and breathing. The fundamental cause of ALS is unknown and as a result treatment options are primarily restricted to physical therapy to help prolong muscle strength....

Published: 3/14/2025 | Inventor(s): Philip Wong, Aneesh Donde, Jonathan Ling, Liam Chen, Mingkuan Sun

Keywords(s): Amyotrophic Lateral Sclerosis, CNS and Neurological Disorders, Disease Indication, Target, Therapeutic Matter, Therapeutics

Category(s): Clinical and Disease Specializations > Neurology > Amyotrophic Lateral Sclerosis, Technology Classifications > Therapeutic Modalities > Targets

TDP-43 Suppression of Non-conserved Cryptic Exons is Compromised in Human Neurodegenerative Diseases

C13621: Suppression of Non-conserved Cryptic Exons is Compromised in Human Neurodegenerative DiseasesAbstractA novel mechanism underlying the suppression of non conserved cryptic exons. This mechanism is essential to maintain intron integrity, and is compromised in ALS-FTD, indicating that loss of function underlies pathogenesis of disease. Publication...

Published: 3/13/2025 | Inventor(s): Philip Wong, Jonathan Ling

Keywords(s): Alzheimer's Disease, Amyotrophic Lateral Sclerosis, Basic Research Biomarker, CNS and Neurological Disorders, Dementia, Discovery/Research Tools, Disease Indication, In Vivo Research Tool, Knock Out Mouse Model, Mouse Model

Category(s): Clinical and Disease Specializations, Clinical and Disease Specializations > Neurology, Technology Classifications > Research Tools > Animal Models, Technology Classifications > Research Tools, Clinical and Disease Specializations > Neurology > Alzheimer's Disease, Clinical and Disease Specializations > Neurology > Amyotrophic Lateral Sclerosis, Clinical and Disease Specializations > Neurology > Dementia

NG2-cDTa mice

C11678: Transgenic Mouse Line with Inducible Diphtheria Toxin GeneNovelty: A novel line of transgenic mice expressing stable inducible diphtheria toxin gene under the control of an NG-2 promoter. This mouse line can be used in neuroscience studies to induce a model state of chronic demyelination.Value Proposition: Neuronal cell studies are an important...

Published: 3/13/2025 | Inventor(s): Shin Kang, Dwight Bergles

Keywords(s): Amyotrophic Lateral Sclerosis, Amyotrophic Laterial Sclerosis (ALS), Basic Research Biomarker, CNS and Neurological Disorders, Discovery/Research Tools, Disease Indication, In Vivo Research Tool, Knock In Mouse Model, Motor Neuron Diseases, Mouse Model, Mouse Model Xenograft, Neurodegeneration

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