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COMPOSITIONS FOR TREATING NEURODEGENERATIVE DISEASES AND METHODS THEREOF

NºPublicación:  WO2025245643A1 04/12/2025
Solicitante: 
RAYA THERAPEUTIC INC [CA]
RAYA THERAPEUTIC INC
WO_2025245643_PA

Resumen de: WO2025245643A1

Disclosed herein are compounds and methods of use thereof effective for the treatment of neurodegenerative diseases such as amyotrophic lateral sclerosis (ALS), a disease that affects nerve cells in the brain and spinal cord, eventually causing loss of muscle strength. The compounds of the disclosure include, cutamesine, a synthetic sigma receptor agonist selective for the 81 receptor, and also a chaperone protein of the central nervous system that plays a key role in the modulation of calcium ions and apoptosis, as well as a sapogenin, such as smilagenin, a non-peptide neurotrophic factor that aids in the reversal of free radical neurotoxicity.

HSPE1 Pharmaceutical composition for treating Parkinson's disease containing cell-transducing HSPE1 fusion protein

NºPublicación:  KR20250169645A 04/12/2025
Solicitante: 
한림대학교산학협력단
KR_20250169645_PA

Resumen de: KR20250169645A

과제: 부작용이 적거나 없으며, 효과가 우수한 파킨슨병 예방 또는 치료용 약제를 제공하는 것. 해결수단: 본 발명자들은 HSPE1에 단백질 수송 도메인을 함께 융합시켜 세포 또는 조직으로의 침투를 가능하게 하여, HSPE1 융합단백질이 MPP+와 MPTP로 유도한 도파민성 세포사멸과 파킨슨 동물 질환 모델에서 세포 보호효능을 발현하는지를 연구하였다. 그 결과, 세포 투과성 HSPE1 융합단백질은 파킨슨병에서 효과적인 단백질 치료제로서의 가능성이 있음을 확인하였다.

SCYLLO-INOSITOL IN COMBINATION WITH IMMUNOTHERAPEUTICS FOR THE TREATMENT OF ALZHEIMER'S DISEASE

NºPublicación:  MX2025002727A 01/12/2025
Solicitante: 
EIRGEN PHARMA LTD [IE]
EIRGEN PHARMA, LTD
CN_120826223_PA

Resumen de: MX2025002727A

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 lecanemab are disclosed as useful.

Aav treatment of huntington's disease

NºPublicación:  IL323940A 01/12/2025
Solicitante: 
UNIV OF MASSACHUSETTS [US]
UNIVERSITY OF MASSACHUSETTS
IL_323940_A

Resumen de: ZA201902226B

Aspects of the disclosure relate to compositions and methods useful for treating Huntington' s disease. In some embodiments, the disclosure provides interfering nucleic acids (e.g., artificial miRNAs) targeting the huntingtin gene (HTT) and methods of treating Huntington's disease using the same.

RNAI AGENTS FOR INHIBITING EXPRESSION OF ATAXIN-2 (ATXN2), COMPOSITIONS THEREOF, AND METHODS OF USE

NºPublicación:  MX2025010867A 01/12/2025
Solicitante: 
ARROWHEAD PHARMACEUTICALS INC [US]
ARROWHEAD PHARMACEUTICALS, INC
AU_2024240456_A1

Resumen de: MX2025010867A

Described are RNAi agents, compositions that include RNAi agents, and methods for inhibition of a Ataxin-2 (ATXN2) gene. The ATXN2 RNAi agents and RNAi agent conjugates disclosed herein inhibit the expression of an ATXN2 gene. Pharmaceutical compositions that include one or more ATXN2 RNAi agents, optionally with one or more additional therapeutics, are also described. Delivery of the described ATXN2 RNAi agents to central nervous system (CNS) tissue, in vivo, provides for inhibition of ATXN2 gene expression and a reduction in ATXN2 activity, which can provide a therapeutic benefit to subjects, including human subjects, for the treatment of various diseases including spinocerebellar ataxia type 2 (SCA2) or amyotrophic lateral sclerosis (ALS.)

VARIANT RNAi

NºPublicación:  CA3255657A1 01/12/2025
Solicitante: 
GENZYME CORP [US]
Genzyme Corporation
AU_2025205501_A1

Resumen de: CA3255657A1

Provided herein are RNAi molecules including a first strand containing a guide sequence and a second strand comprising a non-guide sequence where the non-guide sequence contains a bulge opposite the seed region of the guide sequences; e.g., opposite the cleavage sequence. In some aspects, the invention provides RNAi 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.

PREVENTIVE OR THERAPEUTIC AGENT FOR NEURODEGENERATIVE DISEASE

NºPublicación:  MX2025013089A 01/12/2025
Solicitante: 
MEDILABO RFP INC [JP]
UNIV PUBLIC CORPORATION OSAKA [JP]
MEDILABO RFP, INC,
UNIVERSITY PUBLIC CORPORATION OSAKA
WO_2024228392_PA

Resumen de: MX2025013089A

The purpose of the present invention is to provide a drug that exhibits the effect of inhibiting aggregation of a causative protein of an HRE-related neurodegenerative disease such as ALS. According to the present invention, rifampicin or a related substance selected from the group consisting of rifampicin, a derivative thereof, and salts thereof, and/or resveratrol or a related substance selected from the group consisting of resveratrol and a derivative thereof is an active ingredient of a preventive or therapeutic agent for a neurodegenerative disease caused by TDP-43 accumulation, or an active ingredient of a preventive or therapeutic agent for ALS.

TREATMENT OF PARKINSON'S DISEASE IN A PATIENT USING A GLUCOCEREBROSIDASE ACTIVATOR

NºPublicación:  MX2025010647A 01/12/2025
Solicitante: 
BIAL R&D INVEST S A [PT]
BIAL - R&D INVESTMENTS, S.A
AU_2024237252_A1

Resumen de: MX2025010647A

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.

Preparation of CD73 positive NK cells of umbilical cord blood from different starting sources and application of CD73 positive NK cells in treatment of Alzheimer's disease

NºPublicación:  CN121015869A 28/11/2025
Solicitante: 
SHENZHEN ZHONGJIA BIOMEDICAL TECH CO LTD
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CN_121015869_PA

Resumen de: CN121015869A

The invention belongs to the technical field of medicines, and particularly relates to preparation of CD73 positive NK cells of umbilical cord blood from different starting sources and application of the CD73 positive NK cells to treatment of Alzheimer's disease. The CD73 + NK cells differentiated from different initial cell types (CD34 positive cells, CD3 negative/CD56 positive cells and CD56 negative cells) of umbilical cord blood are studied, it is found that all the initial cell types of umbilical cord blood sources can be differentiated into the CD73 + NK cells, 34 +-73-NK cells show a higher growth rate, 56-73-NK cells show higher cytotoxicity, and the CD73 + NK cells can be differentiated into the CD73 + NK cells. And the overall effect of the 56-73-NK cell is superior to that of the 34 < + >-73-NK cell. Further research shows that the CD73 + NK can inhibit activation of STZ injection mouse hippocampal microglia and astroglia, so that the neuroinflammation of the hippocampal is reduced, and a new solution is expected to be provided for treatment of AD.

Robinine with neuroprotective effect and preparation method thereof

NºPublicación:  CN121021509A 28/11/2025
Solicitante: 
ZHEJIANG NORMAL UNIV XINGZHI COLLEGE
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CN_121021509_PA

Resumen de: CN121021509A

The invention relates to the technical field of ormosine compounds, discloses ormosine with a neuroprotective effect and a preparation method thereof, the ormosine is structurally characterized by having a quinolizidine and quinoline structure mother nucleus, and provides a preparation method of novel compounds 1-4 and application of a compound 3 with a neuroprotective effect. The ormosia henryi alkali compound is extracted and separated from ormosia henryi, the compound 3 has a remarkable protection effect on PC12 cell apoptosis induced by A beta 25-35, EC50 is 15.49 mu M, and the ormosia henryi alkali compound can be used for developing drugs for resisting neurodegenerative diseases such as Alzheimer's disease or Parkinson's disease and has a wide application prospect.

2-substituted-4-aminoquinoline derivative as well as preparation method and application thereof

NºPublicación:  CN121021496A 28/11/2025
Solicitante: 
CHENGDU UNIV OF TECHNOLOGY
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CN_121021496_PA

Resumen de: CN121021496A

The invention belongs to the field of medicinal chemistry, and discloses 2-substituted-4-aminoquinoline derivatives as shown in formulas I and II. Wherein R is selected from-OMe and-OH; r1 is selected from the group consisting of thiophen-2-yl and p-meta-phenyl, and R2 is selected from the group consisting of thiophen-2-yl and p-meta-phenyl; r2 is selected from-CH3,-F,-H and-Ph. The raw materials of the 2-substituted-4-aminoquinoline derivative are easy to obtain, the synthesis method is simple, and the operation is simple and convenient. The 2-substituted-4-aminoquinoline derivative has good acetylcholinesterase inhibitory activity, the activity of an individual compound is higher than that of a clinical drug rivastigmine, and the 2-substituted-4-aminoquinoline derivative is expected to be developed into a novel cholinesterase inhibitor type anti-Alzheimer's disease drug.

Treatment for parkinson’s disease

NºPublicación:  NZ748592A 28/11/2025
Solicitante: 
SUN PHARMA ADVANCED RES COMPANY LIMITED
SUN PHARMA ADVANCED RESEARCH COMPANY LIMITED
US_2024066014_PA

Resumen de: NZ748592A

The invention relates to a method of treating or preventing Parkinson’s disease in a subject comprising administering a compound of Formula I wherein, R1is -NHC(O) C3-6cycloalkyl and R2is hydrogen; or R1 and R2along with the carbon atoms to which they are attached form a six membered aromatic ring, wherein the ring is substituted with one or more groups selected from hydrogen, halogen and C1-6alkyl; R3and R4are independently selected from group comprising hydrogen, halogen, C1-3alkyl, OC1-alkyl, NO3, SC2alkyl, C1-3haloalkyl, OC1-3haloalkyl, and SC1-3haloalkyl; or a pharmaceutically acceptable salt thereof.

Application of miRNA-532-5p in Parkinson's disease treatment and prognosis target drugs

NºPublicación:  CN121023005A 28/11/2025
Solicitante: 
NANFANG HOSPITAL OF SOUTHERN MEDICAL UNIV
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CN_121023005_PA

Resumen de: CN121023005A

The invention provides an application of miRNA-532-5p (micro Ribonucleic Acid-532-5p) in a Parkinson's disease treatment and prognosis target drug. In the invention, firstly, it is verified that after overexpression of miRNA-532-5p, the athletic ability of mice is obviously recovered, and the TH content of striatum and nigra is obviously increased compared with that of MPTP group mice; secondly, after the miRNA-532-5p is over-expressed, the mRNA level of an inflammatory factor is obviously reduced, and the mRNA level of an anti-inflammatory factor is obviously increased; and finally, after the miRNA-532-5p is over-expressed, the content of serum inflammatory factors is obviously reduced, and the content of anti-inflammatory factors is obviously increased. In conclusion, the miRNA-532-5p simulant can enhance the function of the miRNA-532-5p, so that the neuroinflammation is relieved, the condition of the Parkinson's disease is relieved, and a new strategy is provided for clinical treatment of the Parkinson's disease.

Regeneration of functional neurons for treatment of neurological disorders

NºPublicación:  CN121015915A 28/11/2025
Solicitante: 
THE PENN STATE RES FOUNDATION
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CN_121015915_A

Resumen de: CN121015915A

The present application relates to regenerating functional neurons for the treatment of neurological disorders. The present document provides methods and materials involved in the treatment of mammals having a cranial nerve disorder, such as Alzheimer's disease (e.g., Alzheimer's disease). For example, methods and materials are provided for administering a composition comprising an exogenous nucleic acid encoding a NeuroD1 polypeptide to a mammal suffering from a cranial nerve disorder.

Application of tranquilizing composition in preparation of medicine for preventing or treating Alzheimer disease

NºPublicación:  CN121015801A 28/11/2025
Solicitante: 
UNIV QINGHAI
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CN_121015801_PA

Resumen de: CN121015801A

The invention belongs to the field of traditional Chinese medicine, and particularly relates to application of a tranquilizing composition in preparation of a medicine for preventing or treating Alzheimer's disease, the tranquilizing composition has a remarkable treatment effect on CL4176 nematodes, and has a remarkable inhibition effect on paralysis phenotypes caused by intramuscular A beta overexpression of the CL4176 nematodes. The nerve soothing composition can obviously delay the paralysis phenotype of the Alzheimer's disease caenorhabditis elegans, compared with a positive drug memantine hydrochloride, the paralysis inhibiting drug effect is obviously improved, and the nerve soothing composition has an obvious treatment effect on CL2355 nematodes, has an obvious recovery effect on chemotactic dysfunction of CL2355, has an obvious nerve protection effect, and can be used for treating the CL2355 nematodes. The medicinal value is higher.

Pharmaceutical composition for treating cardiovascular blockage diseases and cognitive impairment and new application of pharmaceutical composition

NºPublicación:  CN121015717A 28/11/2025
Solicitante: 
INSTITUTE OF BASIC RES IN CLINICAL MEDICINE CHINA ACADEMY OF CHINESE MEDICAL SCIENCES
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CN_121015717_PA

Resumen de: CN121015717A

The invention discloses a pharmaceutical composition for treating cardiovascular blockage diseases and cognitive impairment and new application thereof.The pharmaceutical composition is a traditional Chinese medicine compound heart-tonifying and mental-tranquilizing formula, and the compound has the effects of tonifying qi, activating blood, dispersing blood stasis, replenishing essence, nourishing yin and the like; the traditional Chinese medicine composition can be used for integrally intervening a plurality of pathological links of cardiovascular blockage diseases such as myocardial infarction and cognitive impairment diseases such as Alzheimer's disease, not only can improve the functions of the heart and the brain, but also can obviously improve the life quality of a patient, achieves a good clinical curative effect, and does not have obvious adverse reaction.

Pharmaceutical composition for treating degenerative diseases and application thereof

NºPublicación:  CN121015894A 28/11/2025
Solicitante: 
JILIN DASHU BIOTECHNOLOGY CO LTD
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CN_121015894_PA

Resumen de: CN121015894A

The invention relates to a pharmaceutical composition for treating degenerative diseases and application thereof. The pharmaceutical composition is particularly suitable for preventing and treating Alzheimer's disease (AD). The pharmaceutical composition disclosed by the invention comprises a combination of a PDE4 inhibitor and a PDE10A inhibitor, and can be used for remarkably activating a cAMP-PKA-CREB signal channel, enhancing the stress tolerance of neurons, reducing the pathological accumulation of A beta and p-tau and improving the plasticity of nerves. The invention provides an innovative medicine composition with potential clinical application value, and a new strategy is provided for intervention of neurodegenerative diseases.

Application of medicine prepared based on vascular endothelial repair and containing cryptochlorogenic acid in Alzheimer disease

NºPublicación:  CN121015623A 28/11/2025
Solicitante: 
NINGBO MEDICAL TREATMENT CENTER LIHUILI HOSPITAL NINGBO UNIV LIHUILI HOSPITAL
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CN_121015623_PA

Resumen de: CN121015623A

The invention aims to apply cryptochlorogenic acid as a raw material to senescence, migration and tubulation of vascular endothelial cells based on vascular injury pathology, and is used for treating Alzheimer's disease. The Alzheimer disease medicine containing cryptochlorogenic acid is prepared based on vascular endothelial repair, the active ingredient in the medicine is cryptochlorogenic acid, and the medicine is used for inhibiting senescence of vascular endothelial cells, recovering migration ability of the vascular endothelial cells and recovering tubulation ability of the vascular endothelial cells; inhibiting hippocampus CA1 cell atrophy of the animal model with Alzheimer's disease; the damage of Alzheimer's disease serum to a vascular endothelial cell barrier is inhibited.

Application of bitter gourd exosome in regulating intestinal flora

NºPublicación:  CN121015719A 28/11/2025
Solicitante: 
XUZHOU MEDICAL UNIV
XUZHOU AIWEI BIOTECHNOLOGY CO LTD
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CN_121015719_PA

Resumen de: CN121015719A

The invention discloses application of bitter gourd exosomes in regulating intestinal flora. The method comprises the following steps: establishing an Alzheimer's disease mouse model, injecting the extracted balsam pear exosome into the mouse body in a gavage manner to serve as an experimental group drug, taking normal saline as a model negative control group, detecting the change of the intestinal flora of the AD mouse by applying a 16S amplicon sequencing principle, and detecting the change of the intestinal flora metabolite of the AD mouse by applying an LS-MS technology. An open field experiment, a nesting experiment and a water maze experiment are adopted to verify the treatment effect of the balsam pear exosome on AD mouse intestinal flora and metabolic level changes thereof. Results prove that the bitter gourd exosome extracted by the invention can effectively improve the intestinal flora micro-ecology of AD mice; the compound can be used for preparing a microecological preparation for regulating intestinal flora of neurodegenerative diseases such as Alzheimer's disease, Parkinson's disease, Huntington's disease, amyotrophic lateral sclerosis, different types of spinal cerebellar ataxia and the like and diseases such as cerebral apoplexy, cerebral injury, epilepsy, tumor and the like.

Diagnosis and treatment integrated brain-targeted magnetic particle imaging tracer as well as preparation method and application thereof

NºPublicación:  CN121015917A 28/11/2025
Solicitante: 
WENZHOU MEDICAL UNIV
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CN_121015917_PA

Resumen de: CN121015917A

The invention relates to the technical field of biological medicine, in particular to a diagnosis and treatment integrated brain-targeted magnetic particle imaging tracer and a preparation method and application thereof. The preparation method comprises the following steps: loading tetrahydrocurcumin into glucan by adopting a nano-particle self-assembly technology to form glucan nano-particles loaded with tetrahydrocurcumin, synthesizing Fe3O4 nano-particles by adopting a chemical precipitation method, and embedding the Fe3O4 nano-particles on the glucan nano-particles to obtain the diagnosis and treatment integrated brain-targeted magnetic particle imaging tracer agent. The nano-particles are marked as THC (at) Dex-Fe3O4 nano-particles. The THC-Dex-Fe3O4 nanoparticles play a role in treating the Alzheimer's disease during brain imaging diagnosis, and the technical problems that magnetic particle imaging cannot be applied to diagnosis of the Alzheimer's disease and the Alzheimer's disease is difficult to treat are solved.

Tet1-modified siBACE1-entrapped cationic liposome as well as preparation method and application thereof

NºPublicación:  CN121015562A 28/11/2025
Solicitante: 
WEST CHINA HOSPITAL SICHUAN UNIV
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CN_121015562_PA

Resumen de: CN121015562A

The invention discloses a Tet1 modified cationic liposome entrapped with siBACE1 as well as a preparation method and application thereof, and belongs to the technical field of biological medicines. The invention solves the problem of limited treatment effect of siBACE1 for treating Alzheimer's disease in the prior art. The preparation comprises siBACE1, the siBACE1 is coated with a lipid composition, and the lipid composition is modified with a Tet1 peptide; the lipid composition is prepared from 1, 2-dioleoyl-3-trimethyl ammonium propane, dioleoyl phosphatidyl ethanolamine, cholesterol, and distearoyl phosphatidyl ethanolamine-polyethylene glycol 2000, and is characterized in that the lipid composition is prepared from distearoyl phosphatidyl ethanolamine and polyethylene glycol 2000. According to the invention, the siBACE1 can be conveyed to an action site through the Tet1-modified cationic liposome entrapped with the siBACE1, so that the action effect of the siBACE1 is improved, and the siBACE1 can be effectively used for treating the Alzheimer's disease.

Pharmaceutical composition and application thereof

NºPublicación:  CN121015646A 28/11/2025
Solicitante: 
UNIV HUBEI TECHNOLOGY
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CN_121015646_A

Resumen de: CN121015646A

The invention provides a pharmaceutical composition and application thereof, and relates to the technical field of medicine preparation. The pharmaceutical composition comprises S-(-)-cotinine or a pharmaceutically acceptable salt of the S-(-)-cotinine, and rivastigmine or a pharmaceutically acceptable salt of the rivastigmine. Compared with a single drug, the pharmaceutical composition containing S-(-)-cotinine and rivastigmine provided by the invention shows remarkable synergistic interaction and multi-target comprehensive intervention advantages, provides a new choice with better curative effect and more comprehensive action mechanism for treating neurodegenerative diseases such as Alzheimer's disease, and has a good application prospect.

1,4-DIHYDROBENZODPYRAZOLO3,4-F1,3DIAZEPINE DERIVATIVES AND RELATED COMPOUNDS AS LRRK2, NUAK1 AND/OR TYK2 KINASE MODULATORS FOR THE TREATMENT OF E.G. AUTOIMMUNE DISEASE

NºPublicación:  US2025361237A1 27/11/2025
Solicitante: 
ORIGENIS GMBH [DE]
Origenis GMBH
US_2025361237_PA

Resumen de: US2025361237A1

The present invention relates to compounds of formula (I) that are capable of modulating, e.g., inhibiting or activating, one or more kinases, especially LRRK2 and/or NUAK1 and/or TYK2 or mutants thereof. The compounds are useful for treating diseases, such as autoimmune diseases, inflammatory diseases, bone diseases, metabolic diseases, neurological and neurodegenerative diseases, cancer, cardiovascular diseases, allergies, asthma, Alzheimer's disease, Parkinson's disease, skin disorders, eye diseases, infectious diseases and hormone-related diseases. The present description discloses the synthesis and characterisation of exemplary compounds as well as pharmacological data thereof (e.g. pages 40 to 146; examples 1 to 63; compounds 1 to 248; tables 1 to 3). Preferred compounds are e.g. 1,4-dihydrobenzodpyrazolo3,4-f1,3diazepine derivatives and related compounds. An exemplary compound is e.g. 5-(2,6-difluorophenyl)-8-methoxy-1,4-dihydrobenzodpyrazolo3,4-f1,3diazepine (example 49). (Formula (II):

TREATMENT REGIMENS FOR EARLY IDIOPATHIC PARKINSON'S DISEASE

NºPublicación:  AU2023449890A1 27/11/2025
Solicitante: 
BIAL PORTELA & CA S A
BIAL-PORTELA & CA., S.A
AU_2023449890_A1

Resumen de: AU2023449890A1

Opicapone for use as adjunctive therapy to preparations of levodopa and a DOPA decarboxylase inhibitor (DDCI) in the treatment of Parkinson's disease; characterised in that a patient with Parkinson's disease is treatable with preparations of levodopa and a DDCI without clinically diagnosed motor complications.

METHODS AND COMPOSITIONS FOR REDUCING SYMPTOMS OF PARKINSON'S DISEASE

Nº publicación: AU2024250257A1 27/11/2025

Solicitante:

NEURODERM LTD
NEURODERM, LTD

AU_2024250257_A1

Resumen de: AU2024250257A1

Disclosed is a method for the treatment of a neurological or movement disorder, e.g., Parkinson's disease, in a patient in need thereof, by parenteral administration of levodopa and a dopa decarboxylase inhibitor (DDCI), such as carbidopa, benserazide or any combination thereof.

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