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161
resultados
Última actualización
26/06/2026 [09:55:00]
Resumen de: AU2024399699A1
SnRNA systems targeting SOD1 RNA sequences are disclosed herein. Further disclosed are methods of treating Amyotrophic Lateral Sclerosis.
Resumen de: AU2024406255A1
Compositions and methods are disclosed herein for the treatment of Alzheimer's disease with allogeneic mesenchymal stem cells. The methods of treatment involve the administration of a composition of allogeneic mesenchymal stem cells to a subject in need thereof, wherein the efficacy of the treatment methods can be determined through the measurement of specific biomarkers and improved cognitive function and/or quality of life.
Resumen de: AU2024379023A1
Provided herein are expression cassettes for expressing a transgene in a cell, wherein the transgene encodes a GCase polypeptide. Also provided are methods to treat Gaucher Disease or GBA-PD. Further provided herein are vectors (e.g., rAAV vectors), viral particles, pharmaceutical compositions, and kits for expressing an GCase polypeptide in an individual in need thereof.
Resumen de: AU2023477048A1
The present invention relates to pharmaceutical compositions and medicaments comprising the compound of formula (I); or a stereoisomer, tautomer, pharmaceutically usable solvate or salt thereof, and dosage regimens for the administration thereof to human patients.
Resumen de: EP4763987A2
Provided herein are RNAi molecules for treating neurodegenerative synucleinopathies. In some embodiments, the RNAi molecules target expression of alpha-synuclein (SNCA). Further provided herein are expression constructs, vectors (e.g., rAAV), cells, viral particles, and pharmaceutical compositions containing the RNAi. Yet further provided herein are methods and kits related to the use of the RNAi, for example, to treat neurodegenerative synucleinopathies including Parkinson's disease, multiple system atrophy, and dementia with Lewy bodies.
Resumen de: EP4763164A2
0001 The invention features sublingual film formulations of dopamine agonists and methods of treating Parkinson's disease, tremors, restless leg syndrome, sexual dysfunction, and depressive disorders therewith.
Resumen de: WO2025038863A1
Disclosed is a compound represented by Formula (I) or a pharmaceutically acceptable salt thereof. The variables in Formula (I) are defined herein. Compounds of Formula (I) are useful for regulating GPR17 activity and for treating disorders and diseases mediated by GPR17 in humans or non-humans.
Resumen de: WO2025038979A1
Material compositions and/or methods useful for the prophylaxis and/or treatment of protein depletion (proteinopenia) are provided, including material compositions that retains native function of a peptide/protein while limiting and/or preventing amyloid formation and/or aggregation of said peptide/protein. Material compositions and formulations for enhancing peptide/protein solubility, stability, circulation time, receptor interaction, brain penetrance, CSF half-life, facilitating peptide/protein synthesis and purification are also provided.
Resumen de: MX2026001933A
The present invention relates to novel compounds, to said compounds for use as a medicine, more in particular for the prevention or treatment of diseases mediated by activity of cGAS, yet more in particular for the prevention or treatment of inflammation conditions or cancer. The present invention also relates to a method for the prevention or treatment of said diseases comprising the use of the novel compounds. The present invention furthermore relates to pharmaceutical compositions or combination preparations of the novel compounds as well as to said compositions or preparations for use as a medicine, more preferably for the prevention or treatment of diseases mediated by activity of cGAS, yet more in particular for the prevention or treatment of inflammation conditions or cancer. The present invention also relates to processes for the preparation of said compounds.
Resumen de: EP4763834A2
The disclosure provide compounds of Formula Iand the pharmaceutically acceptable salts thereof. The variables, R1, R2, R3, X1, X2, and Z are defined herein. Certain compounds of Formula I act as selective mitochondrial protonophore uncouplers that do not affect the plasma membrane potential. Compounds and salts of Formula I are useful for treating or decreasing the risk of conditions responsive to mitochondrial uncoupling, such as cancer, obesity, type II diabetes, fatty liver disease, insulin resistance, Parkinson's disease, ischemia reperfusion injury, heart failure, non-alcoholic fatty liver disease (NALFD), and non-alcoholic steatohepatitis (NASH). Because mitochondrial uncouplers decrease the production of reactive oxygen species (ROS), which are known to contribute to age-related cell damage, compounds of Formula I are useful for increasing lifespan. Compounds and salts of Formula I are also useful for regulating glucose homeostasis or insulin action in a patient.
Resumen de: EP4763972A1
0001 Provided are an Akkermansia muciniphila strain, and a product and a use thereof. The Akkermansia muciniphila strain is AKK PROBlO, and the preservation number thereof is CGMCC No. 20955, The AKK PROBIO has good tolerance, high safety, a wide range of indications and a good treatment effect, and can prevent and treat diseases such as colitis. colorectal cancer. Alzheimer's disease, and gouty arthritis.
Resumen de: AU2026204322A1
The disclosure provides methods for treating a subject in need thereof comprising administering to the subject a therapeutically-effective dose of psilocybin. The methods described herein may be used to treat a variety of diseases, disorders, and conditions. For example, the methods may 5 be used to treat neurocognitive disorders (e.g., Alzheimer's disease, Parkinson's disease), ADHD, Epilepsy, Autism, Sleep-wake disorders, Chronic pain, Inflammatory Disorders, IBD, Stroke, ALS, and/or Multiple Sclerosis. un u n
Resumen de: WO2026128833A1
In one aspect the present disclosure provides probiotic compositions having at least one gut bacterial species belonging to one or more genera selected from among Patescibacteria, Saccharimonadia, Saccharimonadales, Muribaculaceae, Saccharimonadaceae, Candidatus Saccharimonas, Lachnoclostridium, and Ruminococcus and methods for treating or preventing cognitive decline and Alzheimer's disease or improving cognitive performance in a subject in need thereof by administering said composition. In another aspect, the present disclosure provides method for treating or preventing Alzheimer's Disease or cognitive decline or improving cognitive function in a subject in need thereof comprising performing a fecal microbiota transplant (FMT) to the subject, wherein the FMT comprises at least one gut bacterial species belonging to one or more genera selected from among Patescibacteria, Saccharimonadia, Saccharimonadales, Muribaculaceae, Saccharimonadaceae, Candidatus Saccharimonas, Lachnoclostridium, and Ruminococcus.
Resumen de: US20260166178A1
0000 The invention provides that the expression and/or activity of metal ion transporter, such as a Ca<2+>/Mn<2+> transporter and secretory pathway calcium ATPase 1 (SPCA1) or SPCA2 is associated with the restoration and conservation of Golgi integrity in cells, as well as with the restoration of Golgi ion concentrations. Provided herein are methods of restoring or maintaining Golgi integrity in a cell and methods of modulating Golgi ion concentration is a cell including increasing the expression and/or activity of a metal ion transporter such as SPCA1 or SPCA2 protein in the cell. The invention further provides methods of treating neurodegenerative disease, such as amyotrophic lateral sclerosis (ALS). The methods of treatment include increasing the expression and/or activity of a metal ion transporter such as SPCA1 or SPCA2 protein in cells to restore or maintain Golgi integrity and Golgi ion concentration in the cell.
Resumen de: US20260166145A1
The present disclosure provides methods for preventing or treating Alzheimer's disease in a subject, and/or reducing the likelihood or severity of Alzheimer's disease comprising administering to the subject an effective amount of an anti-CD33 antibody or an antigen binding fragment thereof. Alternatively, the methods comprise administering to the subject an effective amount of engineered immune cells expressing a neuronal antigen specific CAR and an anti-CD33 antibody or an antigen binding fragment thereof.
Resumen de: US20260165993A1
Disclosed herein are methods of reducing α-synuclein (α-syn) misfolds or aggregates in the brain, and specifically, to methods of treating neurodegenerative disorders caused by α-synuclein (α-syn) in the brain, also referred to as α-synucleinopathies, and to methods of hindering or preventing α-synucleinopathy misfolds, and/or or to methods of proactively treating individuals at risk of α-synucleinopathy misfolds and associated disease. These α-synucleinopathies refer primarily to Parkinson's disease (PD), dementia with Lewy Bodies (DLB), and/or Multiple system atrophy (MSA). One embodiment includes a method of administering an L-tyrosine compound or derivative thereof to a subject in need thereof, (optionally in combination with an L-serine compound), and reducing the density and/or amount of α-synuclein aggregates in the brain of a mammal, thereby treating a mammal diagnosed with or suffering from or at risk of suffering from one or more α-synucleinopathies.
Resumen de: US20260167636A1
The present invention relates to compounds which are suitable for imaging TDP-43 (Transactive response (TAR) DNA binding protein 43 kDa) aggregates. The compounds can be used, for example, for diagnosing a disease, disorder or abnormality associated with TDP-43 aggregates or a TDP-43 proteinopathy, such as amyotrophic lateral sclerosis (ALS), Alzheimer's disease (AD), Frontotemporal dementia (FTD) and limbic-predominant age-related TDP-43 encephalopathy (LATE).
Resumen de: US20260165992A1
0000 Methods of treating central nervous system diseases with a combination of N-acetylcysteine and glucagon-like peptide-1 receptor agonist are disclosed. The methods include treatment of Parkinson's disease.
Resumen de: US20260166181A1
0000 The present disclosure relates to AAVs encoding a SOD1 targeting polynucleotide which may be used to treat amyotrophic lateral sclerosis (ALS) and delivery methods for the treatment of spinal cord related disorders including ALS.
Resumen de: WO2026128848A1
The invention is a method for treating patients diagnosed with Parkinson's disease (PD), including post-encephalitic parkinsonism, parkinsonism that may follow carbon monoxide intoxication in adults, or parkinsonism that may follow manganese intoxication in adults by orally administering a controlled release muco-adhesive levodopa formulation and the method provides an improvement of a patient's total post-dose "Off" time compared to post-dose of treatment regimens with oral immediate release levodopa tablets administered with one or more COMT inhibitors, and/or FDA approved controlled release CD-LD product.
Resumen de: US20260167604A1
0000 Provided herein are compounds that inhibit HDAC6, a protein whose activity is associated with a variety of diseases (e.g., cancer, neurological disorders). Also provided are pharmaceutical compositions and kits comprising the compounds, and methods of treating HDAC6-related diseases and disorders (e.g., Alzheimer's disease, cancer) with the compounds in a subject, by administering the compounds and/or compositions described herein.
Resumen de: US20260166091A1
The present disclosure provides methods of lineage specific differentiation of pluripotent stem cells, including induced pluripotent stem cells, into floor plate midbrain progenitor cells, determined dopamine (DA) neuron progenitor cells, and/or DA neurons. Also provided are compositions and uses thereof, such as for treating neurodegenerative diseases and conditions, including Parkinson's disease.
Resumen de: US20260166006A1
0000 The present disclosure relates to methods of treating neurological diseases, comprising administering to a subject in need thereof, a therapeutically effective amount of a compound or pharmaceutical composition according to any embodiment described herein. The present disclosure relates to the methods of treating Parkinson's disease and Dementia with Lewy Bodies, comprising administering to a subject in need thereof, a therapeutically effective amount of a compound or pharmaceutical composition according to any embodiment described herein.
Nº publicación: US20260167941A1 18/06/2026
Solicitante:
PREVAIL THERAPEUTICS INC [US]
PREVAIL THERAPEUTICS, INC.
Resumen de: US20260167941A1
The disclosure relates, in some aspects, to compositions and methods for treatment of diseases associated with aberrant lysosomal function, for example Parkinson's disease (PD) and Gaucher disease. In some embodiments, the disclosure provides expression constructs comprising a transgene encoding beta-Glucocerebrosidase (GBA) or a portion thereof alone or in combination with one or more PD-associated genes. In some embodiments, the disclosure provides methods of Parkinson's disease by administering such expression constructs to a subject in need thereof.
BOPI
Sede Electrónica
