Resumen de: US2025230206A1
Inhibitors are provided for targeting γ-secretase to reduce amyloid load as a viable strategy in Alzheimer's disease treatment and drug discovery. γ-secretase has been shown to cleave amyloid precursor protein, causing an increase in the extracellular concentration of amyloid-β peptides. This extracellular concentration increase can lead to a build-up of amyloid plaques in patients and associated health complications for them. The inhibitors bind adjacent the transmembrane domain of amyloid precursor protein through both covalent and non-covalent interactions. These interactions inhibit the ability of γ-secretase to cleave the amyloid precursor protein, halting the build-up of extracellular amyloid plaques. The inhibitors exhibit specificity for amyloid precursor proteins, reducing concerns of potential off-target effects.
Resumen de: US2025230149A1
The present disclosure provides compositions of (4R,5R)-5-(2-chlorophenyl)-4-(5-(phenylethynyl)pyridin-3-yl)oxazo-lidin-2-one (Compound 1). Crystalline and solvate forms of the compound and formulations comprising the compound are also provided. Methods of using the compound and methods of administering the formulations to a subject in need thereof are provided to treat or prevent CNS disorders such as Alzheimer's disease.
Resumen de: US2025228868A1
The present invention relates to the treatment of a sporadic ALS patient with oral fausdil at a dose of 180-240 mg/day. This results in an anticipated 25-50% reduction in the average decline over at least three months as measured using the revised ALS Functional Rating Scale.
Resumen de: WO2024054791A1
The disclosure relates to a combination of active ingredients/adjuvants for the treatment of neurological disorders and diseases such as Alzheimer's disease and mild cognitive impairment (MCI) and memory and cognitive disorders and conditions. In particular, combinations of scyllo-inositol and treatments for Alzheimer's disease such as aducanumab and/or combinations with essential fatty acids such as mixtures of linolenic acid/linoleic acid or vitamin D or vitamin D compounds such as calcifediol are disclosed as useful. The combinations may be in the form of separate dosage forms of each active ingredient or may be an oral dosage form having multiple active ingredients in a single capsule or tablet or oral solution. The invention also relates to a method of treating patients having mild cognitive impairment with MMSE criteria of between 22 to 26 with a pharmaceutically effective amount of scyllo-inositol to treat the disease and to slow down progression to Alzheimer's disease.
Resumen de: GB2637227A
A method of monitoring and treating a subject with ALS based on biomarkers. In some embodiments, the method comprises: identifying that a subject has a ratio of LPS1EGF associated with ALS; and based on the identifying that the subject has the ratio of LPS:EGF associated with ALS, determining that the subject is eligible for sodium chlorite therapy for the ALS, determining based on the ratio of LPS:EGF whether to continue the therapeutic regimen of sodium chlorite; wherein the ratio of LPS:EGF in the subject is no greater than 50 and the therapeutic regimen of sodium chlorite is about 0.2 mg/kg/day to about 3.5 mg/kg/day administered orally and/or parenterally.
Resumen de: US2025223290A1
Novel compounds of Formula (I), and the pharmaceutically acceptable salts thereof, are inhibitors of NLRP3 and may be useful in the treatment, prevention, management, amelioration, control and suppression of diseases mediated by NLPR3. The compounds of the present invention may be useful in the treatment, prevention or management of diseases, disorders and conditions mediated by NLRP3 such as, but not limited to, obesity, gout, pseudogout, CAPS, NASH, MASH, fibrosis, heart failure, idiopathic pericarditis, atopic dermatitis, inflammatory bowel disease, Alzheimer's Disease, Parkinson's Disease, dementia with Lewy bodies (DLB), and traumatic brain injury.
Resumen de: TW202434616A
Compounds, compositions, uses, and methods for increasing cell viability of a dopaminergic neuron, or for preventing or treating dopaminergic neuronal death, are provided herein. In certain examples, methods for reducing symptoms and/or for preventing or treating Parkinson's disease in a subject in need thereof are provided which may include a step of treatment with a GDP-bound form of Rab1a (Rab1a<SP>GDP</SP>), one or more expressible nucleic acids encoding Rab1a<SP>GDP</SP>, or a combination thereof.
Resumen de: AU2023420087A1
Provided herein a method of treating Juvenile Huntington disease in a subject in need thereof comprising orally administering a pharmaceutical composition comprising pridopidine and/or its analog or a pharmaceutically acceptable salt thereof.
Resumen de: US2025223589A1
Disclosed herein are antisense compounds and methods for selectively reducing expression of an allelic variant of a gene containing a single nucleotide polymorphism (SNP). Such methods, compounds, and composition are useful to treat, prevent, or ameliorate diseases, including neurodegenerative diseases, such as Huntington's Disease (HD).
Resumen de: US2025222072A1
Aspects of the disclosure relate to compositions and methods useful for treating Parkinson's disease. In some embodiments, the disclosure provides a method for treating Parkinson's disease comprising administration of a viral vector comprising a GDNF nucleic acid sequence. In some embodiments, administration is locally to the subject putamen. In some embodiments, administration is systemically, e.g., via the viral vector comprising a modified viral capsid, such as for preferentially targeting cells in the CNS or PNS.
Resumen de: AU2023400894A1
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.
Resumen de: EP4582412A1
The present invention relates to: a method for preparing a chiral gamma lactam derivative or a pharmaceutically acceptable salt thereof by using a chiral organocatalytic compound; and a composition for preventing, alleviating or treating muscle diseases, mental diseases, or neurodegenerative diseases, comprising the derivative or the pharmaceutically acceptable salt thereof. The chiral gamma lactam derivative or the pharmaceutically acceptable salt thereof, of the present invention, has an effect of inhibiting MAO-B and MSTN, targets D1-mClu5, and can be used in the prevention, alleviation, or treatment of muscle diseases including sarcopenia, mental diseases including depression, neurodegenerative diseases including Parkinson's disease, and the like.
Resumen de: EP4582144A2
The invention relates to novel monoclonal anti-alpha-synuclein antibodies. The antibodies can be used for treating a synucleinopathy such as Parkinson's disease (including idiopathic and inherited forms of Parkinson's disease), Diffuse Lewy Body Disease (DLBD), Lewy body variant of Alzheimer's disease (LBV), Combined Alzheimer's and Parkinson disease, pure autonomic failure and multiple system atrophy.
Resumen de: US2025213611A1
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.
Resumen de: US2025213564A1
Methods and compositions that treat Alzheimer's disease and other neurodegenerative diseases and/or to ameliorate or improve symptoms associated with Alzheimer's disease. In some aspects, the compositions and methods use a serotonin 4 receptor (5-hydroxytryptamine (serotonin) receptor 4, or 5-HT4R) agonist in combination with: (R,S)-ketamine, a (R,S)-ketamine analog, or a pharmaceutically acceptable salt, derivative, or metabolite thereof; an antagonist of the glutamate N-methyl-D-aspartate (NMDA) receptor (NMDAR); or an agonist of the α-amino-3-hydroxy-5-methyl-4-isoxazolepropionic acid (AMPA) receptor (AMPAR).
Resumen de: US2025214948A1
There is provided herein a compound of formula I,wherein R1, R2, n, X, Q, L, m, R3 and p are as defined herein, which compounds are useful in the treatment of treatment of diseases characterised by impaired signalling of neurotrophins and/or other trophic factors, such as Alzheimer's disease and the like.
Resumen de: US2025214984A1
Disclosed are compounds of following formula (I):in which all symbols have the same meanings as the definitions described in the specification; or a salt thereof. The compounds or a salt thereof are useful as a prophylactic and/or therapeutic agent for cancer, Huntington's disease, Alzheimer's disease and the like.
Resumen de: AU2025204524A1
Abstract 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. Abstract 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. un b s t r a c t u n h e i n v e n t i o n p r o v i d e s n o v e l d o s i n g r e g i m e n s f o r e u c o - e t h y l t h i o n i n i u m ( ) c o m p o u n d s w h i c h m a x i m i s e t h e p r o p o r t i o n o f s u b j e c t s i n w h i c h t h e c o n c e n t r a t i o n w i l l e x c e e d c o n c e n t r a t i o n s i n w h i c h t h e r a p e u t i c e f f i c a c y i n r e l a t i o n t o t r e a t m e n t o f n e u r o d e g e n e r a t i v e d i s o r d e r s s u c h a s l z h e i m e r ' s d i s e a s e a n d r o n t o t e m p o
Resumen de: WO2025141029A1
Disclosed are compounds of formula (I), or a pharmaceutically acceptable salt thereof. Also disclosed are a medicament and a pharmaceutical composition comprising said compounds of formula (I), and said compounds (I) for use in the treatment of a neurodegenerative disease such as Parkinson's disease. Further disclosed are a solid form of a compound of Formula (I-a), characterized as crystalline Form A, as well as a pharmaceutical composition comprising said solid form, and said solid form for use in treating a neurodegenerative disease.
Resumen de: AU2025204414A1
Abstract Provided are compounds, methods, and pharmaceutical compositions for reducing the amount or activity of SNCA mRNA in a cell or animal, and in certain instances reducing the amount of alpha-synuclein protein in a cell or animal. Such compounds, methods, and pharmaceutical compositions are useful to ameliorate at least one symptom or hallmark of a neurodegenerative disease. Such symptoms and hallmarks include motor dysfunction, aggregation of alpha-synuclein, neurodegeneration, cognitive decline and dementia. Such neurodegenerative diseases include Parkinson's disease, dementia withLewy bodies, diffuse Lewy body disease, pure autonomic failure, multiple system atrophy, neuronopathic Gaucher's disease and Alzheimer's disease. Abstract Provided are compounds, methods, and pharmaceutical compositions for reducing the amount or activity of SNCA mRNA in a cell or animal, and in certain instances reducing the amount of alpha-synuclein protein in a cell or animal. Such compounds, methods, and pharmaceutical compositions are useful to ameliorate at least one symptom or hallmark of a neurodegenerative disease. Such symptoms and hallmarks include motor dysfunction, aggregation of alpha-synuclein, neurodegeneration, cognitive decline and dementia. Such neurodegenerative diseases include Parkinson's disease, dementia withLewy bodies, diffuse Lewy body disease, pure autonomic failure, multiple system atrophy, neuronopathic Gaucher's disease and Alzheimer's disease.
Resumen de: AU2024208288A1
Methods of treating a subject having parasomnias related to Rapid Eye Movement (REM) sleep and attenuating disease progression into synucleinopathies and Parkinson's disease by administering a formulation comprising an effective amount of piromelatine to the subject. Methods of treating a subject having parasomnias related to Rapid Eye Movement (REM) sleep and attenuating disease progression of parasomnias into chronic post-traumatic stress disorder (PTSD) by administering a formulation comprising of an effective amount of piromelatine to a subject.
Resumen de: US2025213622A1
The use of extract from rabbit skin inflamed by vaccinia virus for treating Parkinson's disease or restoring neurological function of the brain or alleviating damage to neurological function of the brain in a patient suffering from Parkinson's disease is provided. The extract from rabbit skin inflamed by vaccinia virus can be Lepalvir.
Resumen de: AU2023408627A1
The present invention is directed to the compound golexanolone for use in the treatment of Parkinson's Disease (PD) or for use in the treatment of L-dopa Induced Dyskinesia (LID) in Parkinson's Disease (PD) patients. Further, the present invention is directed to the compound golexanolone or a pharmaceutically acceptable salt thereof, for use in the treatment of Parkinson's Diseases (PD) patients, in particular PD patients exhibiting a L-dopa Induced Dyskinesia (LID).
Resumen de: US2025213507A1
Tramiprosate and derivatives thereof are provided herein for treating neurodegenerative disorders such as Alzheimer's disease (AD).
Nº publicación: US2025213619A1 03/07/2025
Solicitante:
SAISEIKEN CO LTD [JP]
SAISEIKEN CO., LTD
Resumen de: US2025213619A1
A composition for treating intractable neurological diseases contains culture supernatant obtained by culturing deciduous dental pulp stem cells in serum-free medium under ultrasound irradiation. A pharmaceutical product for the treatment of amyotrophic lateral sclerosis (ALS) and Alzheimer's disease contains the composition for the treatment of intractable neurological diseases. In a method for producing a composition for treating intractable neurological diseases, the composition contains a culture supernatant obtained by culturing deciduous dental pulp stem cells under ultrasound irradiation using serum-free medium.