Resumen de: WO2025049807A1
Disclosed are novel strategies for the treatment of patients with Parkinson's disease and other secondary Parkinsonian disorders. Disclosed are DA neuronal cells that have been modified in vitro with a genetic insertion of GDNF. The GDNF coding sequence is inserted under the transcriptional control of a promotor such that secreted proteins are made and taken up by the endogenous cells after the administered engineered cells have matured to a Neuronal Mature Cell Type to promote survival of endogenous neurons. Also disclosed are DA neuronal cells that have been modified with a genetic insertion of GBA in vitro and are hemizygous null for SNCA. The GBA coding sequence is inserted under the transcriptional control of a ubiquitous promotor and the secreted proteins are made and taken up by the graft and endogenous cells immediately upon transplantation to promote long-term graft integrity.
Resumen de: EP4516782A1
The present disclosure relates to novel crystalline forms of Blarcamesine hydrocholoride (hereinafter referred to as "compound I"), a method for preparing same, a pharmaceutical composition comprising the crystalline forms, and use of the crystalline forms in preparing a Sigma-1 receptor agonist and a medicament for treating Rett syndrome, Alzheimer's disease, and Parkinson's disease dementia.
Resumen de: EP4516792A1
The present invention aims to provide a medicament capable of treating and/or preventing diseases associated with oxidative stress by inhibiting the protein-protein interaction between Keap1 and Nrf2 and activating Nrf2. The present invention relates to a compound represented by the following formula (1):wherein each symbol is as described in the DESCRIPTION,or a pharmaceutically acceptable salt thereof. In addition, the present invention also relates to a medicament containing the compound, for the prophylaxis and/or treatment of diseases involving oxidative stress selected from the group consisting of chronic kidney disease, non-alcoholic steatohepatitis, chronic obstructive pulmonary disease, radiation skin disorder, radiation mucosal disorder, cardiac failure, pulmonary arterial hypertension, Parkinson's disease, Friedreich's ataxia, multiple sclerosis, age-related macular degeneration, retinitis pigmentosa and glaucoma.
Resumen de: WO2023209010A1
The present invention generally relates to the treatment of Parkinson's disease (PD) in humans. More particularly, it relates to nicotinamide riboside (NR) for use in a method for treatment of Parkinson's disease in a human subject characterized by a particular dosage regimen; pharmaceutical compositions; and dosage forms, which can be for use in or as treatment of Parkinson's disease.
Resumen de: US2025064879A1
The present invention relates to a composition for treating, improving, or preventing Alzheimer's disease caused by ApoE4 gene mutation, the composition including Banha-Sasim-Tang as an active ingredient.The composition including Banha-Sasim-Tang as an active ingredient of the present invention and a treatment method exhibit excellent effects of reducing amyloid beta proteins and inhibiting deposition thereof, which are specific to Alzheimer's disease caused by ApoE4 gene mutation, thereby having high applicability as a specific composition for preventing, improving, or treating Alzheimer's disease caused by ApoE4 gene mutation.
Resumen de: WO2025041080A1
Provided herein are methods of assessing selenium deficiency in subjects having Huntington's Disease. Further provided herein are methods of treating and/or preventing Huntington's Disease by administering pharmaceutical compositions comprising selenium agents.
Resumen de: US2025064776A1
A method for treating or preventing amyotrophic lateral sclerosis (ALS) or other motor neuron disease in a subject, the method comprising administering to the subject a PKC activating compound (e.g., a bryostatin, such as bryostatin-1, or a bryolog) in a therapeutically effective amount to treat or prevent the motor neuron disease by activating PKC in the subject. The ALS may be, for example, classical ALS, primary lateral sclerosis (PLS), progressive muscular atrophy (PMA), and progressive bulbar palsy (PBP). The PKC activating compound may be administered at an initial loading dose of about 15, 24, or 48 micrograms weekly in the first one week or consecutive two or three weeks, followed by doses of about 12, 20, or 40 micrograms alternately every two or three weeks for at least 4, 5, 6, 8, 10, 12, 14, 16, 18, 20, 24, or 30 total weeks.
Resumen de: US2025066774A1
Disclosed herein are engineered latent guide RNAs targeting LRRK2 and compositions comprising the same for treatment of diseases or conditions (e.g. Parkinson's Disease) in a subject. Also disclosed herein are methods of treating diseases or conditions (e.g. Parkinson's Disease) in a subject by administering engineered latent guide RNAs or pharmaceutical compositions described herein.
Resumen de: AU2025200812A1
Compositions and methods to prevent, inhibit or treat a disease or disorder associated with expression of APOE4 in a mammal are provided.
Resumen de: US2025066336A1
The invention provides novel ligands of Kappa (κ) opioid receptors, such as can be used to modulate a Kappa opioid receptor. Methods of synthesis and methods of use are also provided. Compounds of the invention can be used therapeutically in the treatment of dissociative disorders or pain, or to provide neuroprotection, or to induce diuresis, or to modulate the immune system, or for treatment of one or more of an affective disorders comprising depression or stress/anxiety; an addictive disorder; alcoholism, epilepsy; a cognition deficiency; schizophrenia; Alzheimer's disease; or pain.
Resumen de: US2025066330A1
The invention provides a compound of formula (1), or a pharmaceutically acceptable salt, ester or carbamate thereof, or a salt of such an ester or carbamate,wherein either:R1 is OH, and R2 is H; orR1 is H, and R2 is OH.The invention further provides uses of the compounds of formula (I) and compositions comprising compounds of formula (I), including the use of such compounds for the detection of tau deposits, and the use of such compounds and compositions as diagnostic agents in the diagnosis or monitoring of the progression of a disease or disorder such as Alzheimer's disease, progressive supranuclear palsy and corticobasal degeneration, or for the prevention or treatment of a disease or disorder such as Alzheimer's disease, progressive supranuclear palsy and corticobasal degeneration.
Resumen de: US2025066433A1
The present disclosure provides compositions and methods for modulating transcription of mutant C9orf72 gene alleles in patients in need thereof, including patients having a C9orf72-related disease such as amyotrophic lateral sclerosis (ALS) or frontotemporal dementia (FTD).
Resumen de: US2025066434A1
The present disclosure provides compositions and methods for modulating transcription of mutant C9orf72 gene alleles in patients in need thereof, including patients having a C9orf72-related disease such as amyotrophic lateral sclerosis (ALS) or frontotemporal dementia (FTD).
Resumen de: US2025066364A1
The invention provides substituted pyrazolo1,5-apyrimidine and related organic compounds, compositions containing such compounds, medical kits, and methods for using such compounds and compositions to treat medical disorders, e.g., Gaucher disease, Parkinson's disease, Lewy body disease, dementia, or multiple system atrophy, in a patient. Exemplary substituted pyrazolo1,5-apyrimidine compounds described herein include 5,7-dimethyl-N-phenylpyrazolo1,5-apyrimidine-3-carboxamide compounds and variants thereof.
Resumen de: US2025064970A1
L-N-isobutyryl cysteine (L-NIBC)-bound gold cluster Au18(L-NIBC)14, its use for treating multiple sclerosis, Alzheimer's disease (AD), liver cirrhosis, diabetes, Parkinson's disease (PD) or glaucoma, and the use of it for manufacturing a medicament for treatment of multiple sclerosis, Alzheimer's disease (AD), liver cirrhosis, diabetes, Parkinson's disease (PD) or glaucoma. Composition and methods for treating multiple sclerosis, Alzheimer's disease (AD), liver cirrhosis, diabetes, Parkinson's disease (PD) or glaucoma.
Resumen de: WO2025039918A1
Provided are an Akkermansia muciniphila strain, and a product and a use thereof. The Akkermansia muciniphila strain is AKK PROBIO, 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: US2025066302A1
Deuterated forms of volinanserin according to structural formula (I), and their pharmaceutically acceptable salts, pharmaceutical compositions containing these compounds, and methods of treatment or prevention using these compounds or pharmaceutical compositions are described. The compounds are useful for treating or preventing a disease or condition selected from psychosis, schizophrenia, schizoaffective disorder, Parkinson's disease, Lewy body dementia, sleep disorder (including insomnia), agitation, mood disorder (including depression), thromboembolic disorder, autism, and attention deficit hyperactivity disorder.
Resumen de: WO2025041128A1
The present invention provides dihydroorotate dehydrogenase (DHODH) inhibitors which are brain permeable, and pharmaceutical compositions comprising them. The compounds and pharmaceutical compositions disclosed are useful in preventing, i.e., reducing likelihood of, delaying onset of, or treating diseases or disorders associated with neuronal hyperexcitability, e.g., diseases or disorders which can be treated by the specific inhibition of DHODH enzyme activity such as epilepsy and Alzheimer disease.
Resumen de: WO2025042853A1
Material compositions and/or methods useful for the prophylaxis and/or treatment of a-syn protein depletion (proteinopenia) are provided, including material compositions that retain native function of a-syn protein while limiting and/or preventing amyloid formation and/or aggregation of said protein. Material compositions and formulations for enhancing protein solubility, stability, circulation time, receptor interaction, brain penetrance, CSF halflife, and for facilitating peptide/protein synthesis and purification are also provided.
Resumen de: WO2025042769A1
Described herein are compounds that inhibit poly(ADP-ribose) polymerase (PARP) and pharmaceutical compositions comprising the same. In certain embodiments, the compounds are suitable for treating, ameliorating, and/or preventing cancer. In certain embodiments, the compounds are suitable for treating, ameliorating and/or preventing neurodegenerative diseases and/or disorders, including but not limited to Parkinson's disease, Alzheimer's disease, Lewy body dementia, Alzheimer's disease related dementia or amyotrophic lateral sclerosis.
Resumen de: WO2025040649A1
Described is a pharmaceutical composition comprising an antagonist/inhibitor of neuropeptide B/W receptor (NPBWR1) for use in in a method of treating, ameliorating or preventing a mood disorder/affective disorder and/or chronic stress and/or anxiety disorders and/or Parkinson's disease. Moreover, described is a pharmaceutical composition comprising an agonist/activator of neuropeptide B/W receptor (NPBWR1) for use in in a method of treating, ameliorating or preventing a bipolar affective disorder (ICD-10 F31) during the manic phase, appetitive disorders, preferably anorexia or bulimia. Further, described is a method for assessing the activity of a candidate molecule suspected of being an antagonist/inhibitor or an agonist/activator of NPBWR1.
Resumen de: EP4512413A1
Use of an interleukin 27 (IL-27) protein in preparation of a product for treating and/or delaying Alzheimer's disease (AD) is provided, belonging to the technical field of biomedicine. The IL-27 protein refers to a recombinant IL-27 protein targeting a therapeutic target II,-27, and is selected from the group consisting of a mouse-derived IL-27 protein and a human-derived IL-27 protein, as well as a mammalian IL-27 protein other than the mouse-derived IL-27 protein and the human-derived IL-27 protein. The recombinant IL-27 protein can effectively alleviate the AD caused by Aβ deposition, and can selectively bind to a target receptor, thereby ensuring an accuracy of test results. The protein receptor is highly expressed in the dentate gyrus region of hippocampus, and guarantees drug targeting to the greatest extent. The recombinant IL-27 protein also shows desirable application prospects, can quickly and effectively improve and alleviate behaviors such as the memory impairment in mice with AD, and has a clinical transformation value.
Resumen de: EP4512403A1
Described is a pharmaceutical composition comprising an antagonist/inhibitor of neuropeptide B/W receptor (NPBWR1) for use in in a method of treating, ameliorating or preventing a mood disorder/affective disorder and/or chronic stress and/or anxiety disorders and/or Parkinson's disease. Moreover, described is a pharmaceutical composition comprising an agonist/activator of neuropeptide B/W receptor (NPBWR1) for use in in a method of treating, ameliorating or preventing a bipolar affective disorder (ICD-10 F31) during the manic phase, appetitive disorders, preferably anorexia or bulimia. Further, described is a method for assessing the activity of a candidate molecule suspected of being an antagonist/inhibitor or an agonist/activator of NPBWR1.
Resumen de: EP4512415A1
The present invention relates to a CAR and to regulatory T-cells (Treg) expressing the CAR for use in the treatment of multiple sclerosis (MS) and/or Alzheimers disease (AD). The CAR, especially Treg expressing the CAR, are suitable for suppressing adverse immune reactions occurring in MS and/or AD due to the high specificity of the CAR for myelin basic protein (MBP) or myelin oligodendrocyte glycoprotein (MOG), which are primary targets of autoimmune reactions in MS and AD.
Nº publicación: NL2039691A 24/02/2025
Solicitante:
UNIV JINAN [CN]
JINAN UNIVERSITY
Resumen de: NL2039691A
The invention discloses the application of fibroblast growth factor 17 (FGF17) protein and/or its activators in the preparation of drugs for treating Alzheimer’s disease, belonging to the field of biomedicine technology. This invention is the first to discover that different concentrations of FGF17 can restore neuronal damage caused by Aß, including aspects such as cell viability, cell body condition, and synaptic growth. It plays a significant role in the treatment of Alzheimer’s disease and thus has the potential to become a novel protein-based drug, distinct from existing protein or antibody-based medications.