Alerta

OPTIMISED DOSAGE OF DIAMINOPHENOTHIAZINES IN POPULATIONS

Resumen de: US2025302843A1

The invention provides novel dosing regimens for Leuco-Methylthioninium (LMT) compounds which maximise the proportion of subjects in which the MT concentration will exceed concentrations in which therapeutic efficacy in relation to treatment of neurodegenerative disorders such as Alzheimer's disease and rontotemporal dementias can be achieved, while maintaining a desirable clinical profile. Also provided are LMT-containing dosage units and other compositions.

Methods for Reducing Ataxin-2 Expression

Resumen de: US2025304970A1

Provided herein are methods for decreasing Ataxin-2 mRNA expression. Such methods are useful to ameliorate symptoms of Ataxin-2 associated diseases. Such Ataxin-2 associated diseases include amyotrophic lateral sclerosis (ALS). Such symptoms include loss of motor function, reduced CMAP amplitude, denervation, and loss of motor neurons.

INDAZOLE DERIVATIVE AND PREPARATION METHOD THEREFOR AND USE THEREOF

Resumen de: WO2025201511A1

The present invention relates to an indazole derivative and a preparation method therefor and a use thereof. The indazole derivative can be used for LRRK2 kinase inhibition or for treating Parkinson's disease (PD).

CRYSTALS OF ANTI-HUMAN TAU MONOCLONAL ANTIBODY

Resumen de: WO2025207501A1

Crystals of an antibody that specifically binds to human tau protein phosphorylated at serine 413 are provided, as well as methods of producing such antibody crystals, compositions comprising the crystals, and uses of such compositions for treating a disease, e.g., Alzheimer's disease. Crystals suitable for X-ray diffraction are also provided, and the inventors herein have used such crystals to solve the three-dimensional structure of the antibody to 2.76 Å resolution.

COMPOUNDS AND METHODS FOR MODULATING ALPHA-SYNUCLEIN EXPRESSION

Resumen de: AU2024220937A1

Provided herein are compounds, phannaceutical compositions, and methods of use for reducing the amount or activity of SNCA mRNA in a cell or subject, and in certain instances reducing the amount of alpha-synuclein protein in a cell or subject. Such compounds, pharmaceutical compositions, and methods of use are useful to ameliorate at least one symptom or hallmark of a synucleinopathy. Such synucleinopathies include Parkinson's disease, dementia with Lewy bodies (DLB), diffuse Lewy body disease, Parkinson's disease dementia (PDD), pure autonomic failure, multiple system atrophy (MSA), neuronopathic Gaucher's disease, and Alzheimer's disease.

BIOMARKERS OF AMYOTROPHIC LATERAL SCLEROSIS AND USES THEREOF

Resumen de: AU2024238511A1

The present disclosure relates to biomarkers and uses thereof in methods for selecting a patient diagnosed with amyotrophic lateral sclerosis (ALS) for an ALS therapy. The present disclosure further relates to methods for identifying the severity of ALS in a patient, treating an ALS patient, and monitoring efficacy of an ALS treatment.

COMPOSITIONS OF BIOACTIVE AGENTS

Resumen de: WO2025201405A1

A pharmaceutical composition comprising at least three components selected from the group consisting of Compound A, Compound B, Compound C, and Compound D, and their use in treating a neurodegenerative disease (e.g., Alzheimer's disease) and improving cognition or memory.

METHODS FOR TREATING ALS WITH A RHO KINASE INHIBITOR BASED ON USE OF NEUROFILAMENT LIGHT CHAIN BIOMARKER

Resumen de: WO2025207476A1

A method of treating amyotrophic lateral sclerosis (ALS), includes: determining for a patient diagnosed with ALS, a pre-treatment amount of neurofilament light chain (NfL) in the patient's serum or plasma; orally administering to the patient a first rho kinase inhibitor in a predetermined amount for a predetermined period of time; determining a post-treatment amount of NfL in the patient's serum or plasma; when the post-treatment of amount of NfL is determined to be lower than the pre-treatment amount of NfL, orally administering to the patient a second rho kinase inhibitor in a therapeutically effective amount for treating ALS; and when the post-treatment of amount of NfL is determined to be not lower than the pre-treatment amount of NfL, withholding administration of the second rho kinase inhibitor to the patient.

ANTIBODIES TO AMYLOID BETA

Resumen de: US2025304670A1

Antibody for human amyloid beta. Antibody selectively binds human amyloid beta 42 peptide over human amyloid beta 40 peptide. Antibodies specific for amyloid beta 42 as therapeutic agents for binding amyloid beta 42 peptide and treating conditions associated with amyloidosis, such as Alzheimer's disease.

MULTISPECIFIC BINDING MOLECULES AND METHODS OF USE THEREOF

Resumen de: US2025304709A1

Multispecific binding molecules having a first binding domain targeting a blood brain barrier target and a second binding domain targeting a neuron target, astrocyte target and/or glial cell target, and uses thereof in aiding the treatment of central nervous system diseases including neurodegenerative diseases such as Alzheimer's disease, Huntington's disease, Parkinson's diseases, Progressive Supranuclear Palsy (PSP), Amyotrophic Lateral Sclerosis (ALS), Frontal Temporal Dementia (FTD), autism, catalepsy, encephalitis, migraine, and Tourette's.

GLUCOCEREBROSIDASE (GBA) POLYMER CONJUGATE, PREPARATION METHOD AND USE FOR NANOTECHNOLOGICAL BASED ENZYME REPLACEMENT THERAPY

Resumen de: MX2025006236A

The present invention relates to the medical field, in particular, to a nanotechnological based Enzyme Replacement Therapy, preferably for Parkinson's disease, based on the restoration of lysosomal glucocerebrosidase activity through enzyme-polymer nanoconjugation of GBA, the GBA polymer conjugate for such use, and its manufacturing method.

METHODS FOR TREATING OBSESSIVE COMPULSIVE RELATED DISORDERS, TIC DISORDERS AND GLUTAMATE EXCITOTOXICITY RELATED DISORDERS

Resumen de: MX2025010042A

The present invention provides for methods of treating obsessive-compulsive disorder (OCD) and OCD-related disorders (body dysmorphic disorder, hoarding disorder, trichotillomania (hair-pulling disorder), excoriation (skin-picking) disorder, substance/medication-induced obsessive-compulsive and related disorder, obsessive- compulsive and related disorder due to another medical condition, and other specified and unspecified obsessive-compulsive and related disorders), Tic disorders including Tourette syndrome, autism spectrum disorder (ASD) and glutamate excitotoxicity related disorders including amyotrophic lateral sclerosis (ALS), Parkinson's disease, traumatic brain injury, multiple sclerosis, Huntington's disease, and schizophrenia, comprising the step of administering an effective amount of a histamine type 1 receptor agonist and/or histamine type 3 receptor antagonist, such as betahistine or its pharmaceutically acceptable salts, analogs, metabolites, prodrugs, derivatives, metabolites, co-crystals, modifications, solvates, hydrates, isotopes, tautomers, esters, polymorphs or stereoisomers.

ALPHA-SYNUCLEIN BINDERS AND METHODS OF USE

Resumen de: MX2025009562A

The invention is directed to compounds of Formula (I) or their pharmaceutically acceptable salts, which may be suitable for imaging alpha-synuclein pathology- and hence are useful in binding and imaging alpha-synuclein aggregates in patients with Parkinson's Disease. More specifically, this invention relates to a method of using the compounds of this invention as tracers in positron emission tomography (PET) imaging to study alpha-synuclein in brain <i>in vivo </i>to allow diagnosis of Parkinson's Disease and other neurodegenerative diseases characterized by alpha-synuclein pathology. The invention further relates to a method of measuring clinical efficacy of therapeutic agents for Parkinson's Disease and other neurodegenerative diseases characterized by alpha-synuclein pathology.

PHARMACEUTICALLY ACTIVE PYRAZOLO-PYRIDONE MODULATORS OF DCN1/2-MEDIATED CULLIN NEDDYLATION

Resumen de: MX2025010188A

A DCN1/2-mediated cullin neddylation modulator; a method for treating disorders associated with dysfunctional DCN1 and/or UBC12, Alzheimer's disease, other neurodegenerative diseases, bacterial infections, or viral infections; and a method for treating cancers are provided. The DCN1/2-mediated cullin neddylation modulator includes a compound according to Formula I disclosed herein. The methods include administering to a mammal a therapeutically effective amount of a compound according to Formula I. Also provided herein is a pharmaceutical composition including a therapeutically effective amount of a compound according to Formula I, or a pharmaceutically acceptable salt thereof, and a pharmaceutically acceptable carrier.

SPIRO DERIVATIVES AS M4 ACTIVATORS/MODULATORS AND USES THEREOF

Resumen de: MX2025007982A

The present disclosure provides compounds of Formula I: (l) (I), or an N- oxide thereof, or a pharmaceutically acceptable salt of the compound or the N-oxide, wherein: A, Y, m, n,p, R¹, R², R³, R^3a, R⁴, R⁵, R⁶, R⁷, and Z are as described herein; processes for the preparation of; intermediates used in the preparation of; and compositions containing such compounds, N-oxides, or salts, and their uses for treating M4-mediated (or M4-associated) disorders including, e.g., Alzheimer's Disease, Parkinson's Disease, schizophrenia (e.g., its cognitive and negative symptoms), pain, addiction, and a sleep disorder.

HUMANISED ANTIBODY AGAINST AMYLOID BETA 42

Resumen de: MX2025009326A

The present invention provides a humanised antibody comprising an antigen-binding domain capable of binding specifically to Aβ42 prefibrillar oligomers with β structure, said antigen-binding domain comprising: (iii) a heavy chain variable region (VH) comprising the sequence of SEQ ID NO.1; or (iv) a light chain variable region (VL) comprising the sequence of SEQ ID NO.2; or a combination thereof. Also provided is the use of the antibody in the treatment of an amyloid disease, and particularly Alzheimer's disease, conjugates and pharmaceutical compositions comprising the antibody, and nucleic acid molecules encoding the antibody or a heavy or light chain polypeptide thereof, as well as vectors and host cells comprising such a molecule.

DUAL INHIBITORS FOR THE TREATMENT OF ALZHEIMER'S DISEASE

Resumen de: MX2025010369A

Compounds (I) are provided, where R¹ and R² are H or (C1-C3)-alkyl; X is a linear methylene chain of formula -CH₂_n- with n = 0, 1 or 2, or a biradical from a branched saturated (C2-C4)-alkylene chain; and A is either a C-radical from a non-aromatic polycyclic 6- to 15-membered carbocyclic ring system, or a C-radical from a polycyclic 6- to 15-membered heterocyclic ring system having one or two O, S or N; wherein the C-radicals are unsubstituted or substituted. Compounds (I) are simultaneously inhibitors of soluble epoxide hydrolase and inhibitors of glutaminyl cyclase. Besides, they reduce the levels of pro-inflammatory cytokines in LPS stimulated BV2 cells, display low cytotoxicity, and have good BBB permeability. Thus, they are useful as multitarget compounds for the prevention or treatment of Alzheimer's disease.

Combination of metformin and glibenclamide in the treatment of parkinson's disease

Resumen de: AU2024232317A1

The present invention provides a pharmaceutical composition comprising metformin and glibenclamide for use in the treatment of Parkinson's disease. The invention also comprises a combined administration of metformin and glibenclamide. In a preferred embodiment, the administration is made through oral route.

PYRIDAZINE COMPOUNDS, THEIR PREPARATION, AND THEIR THERAPEUTIC USES

Resumen de: MX2025010782A

The present invention relates to a compound of formula (I) wherein n is 1 or 2, R1 is halogen, (C₁-C₄)alkyl, halo(C₁-C₄)alkyl, (C₁-C₄)alkoxy, halo(C₁-C₄)alkoxy, ethynyl, propargyl, or (C₃-C₆)cycloalkyl, or two R1 form a cyclopentane ring fused to the phenol; R2 and R3 represent H, cyano, ethynyl, propargyl, (C₁-C₄)alkyl, hydroxy(C₁-C₄)alkyl or a halo(C₁-C₄)alkyl, or R2 and R3 form together with the atoms connecting them a (C₅- C₆)carbocyclic ring fused to the pyridazine ring; R4 and R5 form together with N to which they are attached an optionally substituted 3-7 membered monocyclic heterocycloalkyl ring, 8-11 membered bicyclic heterocycloalkyl ring or 7-12 membered bicyclic heterocyclic spiro ring. The present invention also relates to a medicament and a pharmaceutical composition comprising said compound of formula (I), as well as their therapeutic uses, in particular as inhibitor of NOD-like receptor protein 3 inflammasome for treating for example Parkinson's disease or frontotemporal Dementia.

ALPHA-SYNUCLEIN BINDERS AND METHODS OF USE

Resumen de: MX2025010406A

The invention is directed to compounds of Formula I (I) or their pharmaceutically acceptable salts, which may be suitable for imaging alpha-synuclein pathology and hence are useful in binding and imaging alpha-synuclein aggregates in patients with Parkinson's Disease. More specifically, this invention relates to a method of using the compounds of this invention as tracers in positron emission tomography (PET) imaging to study alpha-synuclein in brain <i>in vivo</i> to allow diagnosis of Parkinson's Disease and other neurodegenerative diseases characterized by alpha-synuclein pathology. The invention further relates to a method of measuring clinical efficacy of therapeutic agents for Parkinson's Disease and other neurodegenerative diseases characterized by alpha-synuclein pathology.

Treatment of parkinson's disease in a patient using a glucocerebrosidase activator

Resumen de: AU2024237252A1

Methods for preventing, limiting or delaying clinical motor progression in a subject with Parkinson's disease with low GCase activity, such as a PD patient with a pathogenic variant in the glucocerebrosidase 1 (GBA1) gene (GBA-PD) is provided, said methods comprising administering a therapeutically effective amount of 5,7-dimethyl-N-((1R,4R)-4- (pentyloxy)cyclohexyl)pyrazolol1,5-apyrimidine-3-carboxamide (Compound A), or a pharmaceutically acceptable salt thereof, to said subject.

1-(CYCLOBUTYLIDENEMETHYL)-2,4,5-TRIMETHOXYBENZENE COMPOUND, PREPARATION METHOD THEREFOR AND USE THEREOF

Resumen de: EP4624448A1

A 1-(cyclobutylidenemethyl)-2, 4, 5-trimethoxybenzene compound and a preparation method and use thereof are provided, belonging to the field of drug development technology. The prepared 1-(cyclobutylidenemethyl)-2, 4, 5-trimethoxybenzene compound may be used for the preparation of an antiepileptic drug and drugs for treatment and/or prevention of traumatic craniocerebral injury disorders, ischemic stroke, hemorrhagic stroke, and Parkinson's disease.

Nanoparticles for use for treating a neuronal disorder

Resumen de: NZ766044A

The present invention relates to the medical field, in particular to the treatment of neurological disorders. More specifically the present invention relates medicaments comprising gold (Au), barium titanate (BaTiO3) and/or zirconium dioxide (ZrO2) nanoparticles coated with a biocompatible coating providing a neutral or a negative surface charge, for preventing or treating Parkinson’s disease or Alzheimer’s disease by intra-cranial or intra-thecal administration and provided without exposure of the nanoparticles to an electric current, electric field or electric stimulus applied by deep brain stimulation (DBS), by transcranial electric stimulation (TES), or by transcranial magnetic stimulation (TMS).

L- Composition for preventing improving or treating Parkinson's disease comprising combination of lactic acid bacteria and L-tyrosine

Resumen de: KR20250141094A

본 발명의 일 예는 락티플란티바실러스 플란타룸(Lactiplantibacillus plantarum) KACC 11451 균주, 락티플란티바실러스 파라플란타룸(Lactiplantibacillus paraplantarum) KACC 12373 균주 또는 락티플란티바실러스 펜토수스(Lactiplantibacillus pentosus) KACC 12428 균주에서 선택되는 1종 이상의 균주 및 L-티로신을 포함하는 파킨슨병 예방, 개선 또는 치료용 조성물을 제공한다. 본 발명의 일 예에 따른 특정 유산균은 L-티로신의 레보도파로의 전환 활성이 매우 높다. 또한, 본 발명의 일 예에 따른 특정 유산균 및 L-티로신을 포함하는 조성물은 생체내에서 레보도파를 생산할 수 있도록 제형화될 수 있고, 파킨슨병의 예방, 개선 또는 치료를 위한 건강기능식품 또는 의약품으로 사용될 수 있다.

VMAT2 INHIBITORS AND METHODS OF USE

Nº publicación: WO2025199234A1 25/09/2025

Solicitante:

NEUROCRINE BIOSCIENCES INC [US]
NEUROCRINE BIOSCIENCES, INC