Absstract of: AU2023427408A1
The disclosure relates to compositions and methods for altering, e.g., enhancing, the expression of GCase proteins, whether in vitro and/or in vivo. Such compositions include delivery of an adeno-associated viral (AAV) particle. The compositions and methods of the present disclosure are useful in the treatment of subjects diagnosed with, or suspected of having Parkinson Disease or related condition resulting from a deficiency in the quantity and/or function of GBA gene product or associated with decreased expression or protein levels of GCase protein.
Absstract of: US2025277040A1
The present invention relates to monoclonal anti-Sortilin antibodies which have been found useful in correcting a deficient level of progranulin (PGRN). In particular, these antibodies can be used in the treatment of frontotemporal dementia (FTD) and amyotrophic lateral sclerosis (ALS).
Absstract of: US2025277203A1
The invention relates to expression vectors, and pharmaceutical compositions, and kits comprising the vectors, and, in particular, their use in methods for treating Parkinson's disease (PD), DOPA responsive dystonia, vascular parkinsonism, side effects associated with L-DOPA treatment for Parkinson's disease, L-DOPA induced dyskinesia, Segawa syndrome, or genetic dopamine receptor abnormalities.
Absstract of: US2025275953A1
The present disclosure relates to a method of treating, or delaying, inhibiting, or suppressing of the progression of, a neurodegenerative disease such as, for example, early-stage Parkinson's disease comprising administering a c-Abl inhibitor (such as vodobatinib, or a pharmaceutically acceptable salt thereof) to a subject in need thereof.
Absstract of: US2025275935A1
Compositions and products (e.g., kits, medical devices, medicaments), which incorporate the compositions, comprising a tetrahydrocannabinol (THC) compound and melatonin compound are provided, Methods for treatment using the compositions and products, methods for diagnosis before or after treatment, and processes for formulating the compositions or manufacturing the products are also provided. They address the need for new treatments of cognitive and perceptual deficits, neuropsychiatric symptoms, neurodegenerative diseases, and neurologic disorders. The inventors discovered therapeutically effective amounts of TI-IC and melatonin can be administered to treat patients, without need for an entourage effect or requirement of cannabidiol (CBD) and other cannabinoid compounds. Optionally, polyphenolic compounds (e.g., curcumin-related compounds, rutin-related compounds) and/or Vitamin E-related compounds (e.g., mixed tocopherols, alpha-tocopherol, beta-tocopherol, gamma-tocopherol) may be administered together with or separately from TI-IC and melatonin compounds.
Absstract of: US2025275936A1
Compositions and methods for treating a cancer or a neurological disease. A method of treating a cancer or a neurological disease comprising administering to a subject in need thereof an effective amount of quercetin, vitamin B3, vitamin C, folic acid, a chemotherapeutic agent, and zafirlukast. A method of treating amyotrophic lateral sclerosis comprising administering to a subject in need thereof an effective amount of quercetin, vitamin B3, vitamin C, folic acid, a chemotherapeutic agent, and zafirlukast.
Absstract of: US2025276011A1
The present disclosure provides, among other things, methods for the treatment of neurodegenerative diseases (ND) and other mitochondrial disorders, and compositions related thereto. Described herein are in vitro (cell culture) and in vivo (animal model) experimental examples demonstrating mitochondrial organelle transplantation (MOT) for the treatment of NDs such as amyotrophic lateral sclerosis (ALS). Furthermore, as discussed herein, MOT has been performed in five human ALS patients with positive results—measurable improvement of their conditions has been observed, with no adverse events.
Absstract of: US2025275995A1
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.
Absstract of: US2025275934A1
Opicapone, levodopa and an AADC inhibitor are described for use in slowing the progression of Parkinson's Disease.
Absstract of: WO2025180180A1
Provided is use of a cell-free fat extract (CEFFE Pro 2.0) in the preparation of a pharmaceutical composition or a health product composition for preventing and/or treating Alzheimer's disease. Treatment of a Caenorhabditis elegans pathology model of Alzheimer's disease with the cell-free fat extract demonstrates that the cell-free fat extract can effectively prolong paralysis of Caenorhabditis elegans of Alzheimer's disease. In a cell experiment, the cell-free fat extract has the effects of promoting the proliferation of SH-SY5Y cells and improving the cell growth state, inhibiting cell agglomeration and shrinkage, and inhibiting cellular damage. The provided cell-free fat extract exhibits potential against Alzheimer's disease and can be used for preparing a pharmaceutical composition or a health product composition for preventing and/or treating Alzheimer's disease.
Absstract of: WO2025181805A1
Provided herein a method for treatment, prevention, alleviation, delaying symptom onset, or slowing of progression of Huntington disease, comprising administering a composition comprising pridopidine or pharmaceutically acceptable salt thereof and at least one composition comprising sigma 2 antagonist agent.
Absstract of: WO2025183416A1
The present invention relates to a composition for treating cognitive dysfunction, specifically Alzheimer's disease. The composition according to one aspect can inhibit or reduce the accumulation of amyloid aggregates and phosphorylated tau aggregates, and improve memory and cognitive function.
Absstract of: WO2025184649A1
The present disclosure provides methods related to treating neurodegeneration and neuroinflammation. In particular, the present disclosure identifies YTH domain-containing family protein 1 (YTHDF1) as a novel therapeutic target for Alzheimer's disease. Embodiments of the present disclosure provide methods of therapy for neurodegenerative disease that involve inhibiting YTHDF1 to induce attenuation of chronic neuroinflammation.
Absstract of: WO2025184593A1
The present disclosure provides methods for preventing or treating a neurodegenerative disorder in a subject, where the neurodegenerative disorder is one characterized by a deposition of abnormal tau protein in the brain of the subject. The method generally includes administering to the subject a therapeutically effective amount of a drug that is a tau propagation antagonist.
Absstract of: WO2025184056A1
Cannabinoid analogs may exhibit anti-inflammatory properties such as by inhibition of cannabinoid type 2 (CB2) receptors. Pharmaceutical compositions comprising the cannabinoid analogs may be used to treat various diseases and conditions in mammals, including pain, anxiety, a sleep disorder, addiction, epilepsy, depression, post-traumatic stress disorder or Alzheimer's disease. In some examples, the pharmaceutical compositions may be administered for treating a cognitive disorder or for improving cognition.
Absstract of: MX2025004703A
Applicant discloses methods and compositions for treating a patient suffering from amyotrophic lateral sclerosis (ALS) comprising administration of a heteroaryl ketone fused azadecalin compound. In embodiments, the heteroaryl ketone fused azadecalin compound is dazucorilant: (R)-(1-(4-fluorophenyl)-6-((4-(trifluoromethyl)phenyl) sulfonyl)-4, 4a, 5,6,7,8-hexahydro-1-H-pyrazolo3,4-gisoquinolin-4a-yl) (pyridin-2-yl)methanone, having the chemical structure illustrated as. Suitable doses include daily administration of 150 milligrams and 300 milligrams of dazucorilant. Suitable doses include daily administration of dazucorilant with food, or with water, or with food and water. Daily administration of dazucorilant is effective to increase dazucorilant exposure up to about 2-fold when continued for seven days or more. Administration of such a heteroaryl ketone fused azadecalin compound may comprise oral administration, enteral administration, or other administration. Pharmaceutical compositions comprising dazucorilant are useful in the treatment of patients suffering from ALS. Suitable pharmaceutical compositions comprising dazucorilant include, e.g., pharmaceutical compositions for oral administration and pharmaceutical compositions for enteral administration.
Absstract of: WO2024092001A1
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.
Absstract of: EP4610260A1
A novel Kv1.3 channel (or KV1.3) inhibitor, which can be used for preventing and/or treating diseases related to the Kv1.3 channel (or Kv1.3), including immune and inflammatory diseases, such as: multiple sclerosis, inflammatory bowel disease, ulcerative colitis, Crohn's disease, rheumatoid arthritis, type I diabetes, psoriasis and asthma, spondylitis, and periodontitis; as well as obesity, type 2 diabetes, renal fibrosis, Alzheimer's disease, ischemic stroke, etc.
Absstract of: MX2025006287A
The present disclosure relates to compositions comprising trehalose and methods of using same for the treatment of Huntington's disease.
Absstract of: MX2025006419A
Provided herein are methods for treating Alzheimer' s disease using a combination of tramiprosate, a tramiprosate prodrug or an active tramiprosate metabolite with at least one amyloid plaque clearing agent.
Absstract of: MX2025008347A
The present disclosure pertains to compositions and methods for the treatment and/or prevention of one or more of obesity, diabetes, metabolic syndrome, Alzheimer's disease, Chronic Fatigue Syndrome (CFS), aging, fibromyalgia, dyslipidemia, hypercholesterolemia, dyslipidemia, Parkinson's disease, migraines, Traumatic Brain Injury (TBI), Attention Deficit Disorder (ADD)/ Attention Deficit Hyperactivity Disorder (ADHD), Cancer, Cardiovascular Disease (CVD)ZCoronary Artery Disease (CAD), Chronic Pain, neuralgia, depression, amyotrophic lateral sclerosis (ALS), and epilepsy, Insufficient Cellular Energy (ICE) and mitochondrial dysfunction. The present disclosure also pertains to methods for increasing mental and/or physical performance levels and/or decreasing exertion during exercise in a subject by the administration of C5 ketones.
Absstract of: MX2025008949A
Provided herein are RNAi molecules for treating Huntington' s disease. Further provided herein are expression cassettes, vectors (e.g., rAAV, recombinant adenoviral, recombinant lentiviral, and recombinant HSV vectors), 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 Huntington's disease.
Absstract of: MX2025009625A
HTT nucleic acid trans-splicing molecules are described, which include a coding domain comprising one or more <i>HTT</i> exons, a splice site, and a binding domain that binds a target intron of a <i>HTT</i> pre-mRNA. HTT nucleic acid trans-splicing molecules described herein may also be used in combination with, e.g., MSH3 binding domains arranged in tandem with an HTT binding domain,<i> </i>MSH3 nucleic acid trans-splicing molecules, <i>MSH3</i> splice modulators, anti-sense oligonucleotides or anti-sense RNA to either of <i>MSH3</i> or <i>HTT</i> and <i>MSH3</i> or <i>HTT</i> microRNA (miRNA) and constructs encoding same. Compositions comprising nucleic acid trans-splicing molecules described herein are also encompassed, as are compositions comprising combinations of nucleic acid trans-splicing molecules with additional therapeutic agents (e.g., <i>MSH3</i> nucleic acid trans-splicing molecules, <i>MSH3</i> splice modulators, anti-sense oligonucleotides or anti-sense RNA to either of <i>MSH3</i> or <i>HTT</i>). Nucleic acid trans-splicing molecules may be used alone or in combination with additional therapeutic agents in methods for treating Huntington's Disease (HD). Nucleic acid trans-splicing molecules are also described herein for use, alone or in combination with additional therapeutic agents, in treating HD or in the preparati
Absstract of: US2024342309A1
Disclosed herein are recombinant gene therapy vectors comprising a PTEN-induced kinase 1 (PINK1) encoding gene that is operatively linked to a promoter and methods of using the recombinant therapy vectors for inhibiting, reducing, or delaying degeneration or death of neurons of a subject.
Nº publicación: MD20250033A2 31/08/2025
Applicant:
HEMPIRE SHANGHAI PHARMACEUTICAL R&D LTD [CN]
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Absstract of: MD20250033A2
Disclosed in the present invention are a cannabinoid composition and an application thereof in the preparation of a drug for treating neurodegenerative diseases, for use in solving the existing problems of large side effects and poorer therapeutic efficacy after long-term use of a drug for treating neurodegenerative diseases. The cannabinoid composition comprises cannabidiol and cannabigerol which are in a mass ratio of 1:1-1:10 or 1:0.3-1:0.5 or 1:0.5-1:0.7 or 1:0.7-1:1. According to the present invention, by using an experimental method for inducing the differentiation of fibroblasts into dopamine nerve cells to observe the morphologies and number of cells after the inducing of cell differentiation and detect an expression level of DA-related factors, and to screen effects of common different cannabinoids and combinations thereof on the inducing of the differentiation of fibroblasts into dopaminergic neurons, it is found that a CBD+CBG combination has optimal effects on the improvement of the survival ability and dopamine secretingability of the dopaminergic neurons, does not contain the addictive substance THC and thus greatly reduces the side effects of a drug, and can be used for preparing a drug for treating neurodegenerative diseases.