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Treatments for amyotrophic lateral sclerosis using dazucorilant

NºPublicación:  IL320512A 01/06/2025
Solicitante: 
CORCEPT THERAPEUTICS INCORPORATED [US]
MANN GRACE [US]
TUDOR IULIA CRISTINA [US]
GUYER WILLIAM [US]
HUNT HAZEL [US]
CUSTODIO JOSEPH [US]
CORCEPT THERAPEUTICS INCORPORATED,
MANN Grace,
TUDOR Iulia Cristina,
GUYER William,
HUNT Hazel,
CUSTODIO Joseph
IL_320512_A

Resumen de: AU2023367284A1

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.

PERCUTANEOUS ABSORPTION PREPARATION CONTAINING DONEPEZIL

NºPublicación:  WO2025110663A1 30/05/2025
Solicitante: 
DAEHWA PHARMACEUTICAL CO LTD [KR]
\uB300\uD654\uC81C\uC57D \uC8FC\uC2DD\uD68C\uC0AC
KR_102704773_B1

Resumen de: WO2025110663A1

Disclosed is a percutaneous absorption preparation containing donepezil that is a pharmacologically active substance useful in treating Alzheimer's dementia symptoms. This percutaneous absorption preparation suppresses donepezil drug crystallization in the percutaneous absorption preparation even when left at room temperature for a long period of time, can improve a percutaneous permeability degree (cumulative permeation amount and skin permeation rate) of the drug, and in particular, can significantly increase the drug absorption of donepezil despite of a small drug loading amount compared to the currently commercially available Donerion patch.

NOVEL KIT OF PHARMACEUTICAL PREPARATIONS FOR THE TREATMENT OF NEURODEGENERATIVE DISEASES

NºPublicación:  US2025170076A1 29/05/2025
Solicitante: 
BERLIREM GMBH [DE]
BERLIREM GMBH
US_2025170076_PA

Resumen de: US2025170076A1

The invention relates to the treatment of neurodegenerative diseases such as Parkinson patients with motor complications in need of Continuous Dopaminergic Stimulation. According to the invention a kit of pharmaceutical preparations is provided comprising levodopa and an AADC-inhibitor. Levodopa is applied via continuous subcutaneous infusion or by an oral modified release formulation and Continuous Dopaminergic Stimulation is achieved by almost complete inhibition of the peripheral levodopa metabolism. To reach far-going blockage of levodopa metabolism, an AADC-inhibitor (e.g., benserazide, carbidopa) is given orally in a much higher than normal dose and, as an indispensable prerequisite, the AADC-inhibitor has to be applied evenly distributed over the day. e.g., via a Modified Release formulation. In order to prevent levodopa conjugation over the COMT pathway, an oral COMT-inhibitor (e.g., opicapone) is also added. In addition, a drug-drug-interaction between the AADC-inhibitor and opicapone further increasing the activity of the former is likely to occur. The resulting effective inhibition of peripheral levodopa metabolism allows a marked reduction of the necessary (equivalent) levodopa (or prodrug) doses to 300-600 mg/day, which is, however, sufficient to reach high levodopa plasma levels of 1500 to 3000 ng/ml. The low daily levodopa dose is infused subcutaneously via minipump and is well-tolerated at the infusion site. Alternatively, the low daily levodopa dose is provid

INHIBITORS OF CYCLIC ADP RIBOSE HYDROLASE METHODS OF USE THEREOF

NºPublicación:  AU2023375532A1 29/05/2025
Solicitante: 
FLAGSHIP PIONEERING INNOVATIONS VI LLC
FLAGSHIP PIONEERING INNOVATIONS VI, LLC
AU_2023375532_A1

Resumen de: AU2023375532A1

The disclosure provides compounds, in part, compounds of Formula I or Formula II and their use in treating medical diseases or disorders, such as neurodegenerative diseases, e.g., Parkinson's disease. Pharmaceutical compositions and methods of making compounds of the disclosure are provided. The compounds are contemplated to be modulators, e.g., inhibitors, of cyclic ADP ribose hydrolase (CD38).

Methods of Treating Alzheimer's Disease

NºPublicación:  US2025170203A1 29/05/2025
Solicitante: 
ATHIRA PHARMA INC [US]
Athira Pharma, Inc
US_2025170203_PA

Resumen de: US2025170203A1

The present disclosure relates to a method of treating mild to moderate Alzheimer's disease comprising administering to a subject with Alzheimer's disease ATH-1017.

INHIBITORS OF CYCLIC ADP RIBOSE HYDROLASE METHODS OF USE THEREOF

NºPublicación:  AU2023375535A1 29/05/2025
Solicitante: 
FLAGSHIP PIONEERING INNOVATIONS VI LLC
FLAGSHIP PIONEERING INNOVATIONS VI, LLC
AU_2023375535_A1

Resumen de: AU2023375535A1

The disclosure provides compounds, in part, compounds of Formula I, Formula II or Formula III and their use in treating medical diseases or disorders, such as neurodegenerative diseases, e.g., Parkinson's disease. Pharmaceutical compositions and methods of making compounds of the disclosure are provided. The compounds are contemplated to be modulators, e.g., inhibitors, of cyclic ADP ribose hydrolase (CD38).

INDOLIN-2-ONE OR PYRROLO -PYRIDIN -2 -ONE DERIVATIVES

NºPublicación:  US2025171439A1 29/05/2025
Solicitante: 
HOFFMANN LA ROCHE INC [US]
Hoffmann-La Roche Inc
US_2025171439_A1

Resumen de: US2025171439A1

The present invention is concerned with 2-oxo-2,3-dihydro-indoles of general formulawhereinAr1 is phenyl or a five or six membered heteroaryl group, containing one, two or three heteroatoms, selected from N, S or O, wherein the N-heteroatom in the heteroaryl group may be oxidized to N+—(O−);R1 is lower alkyl, halogen, cyano or cycloalkyl;Ar2 is a five or six membered heteroaryl group, containing one, two, three or four heteroatoms, selected from N, S or O, wherein the N-heteroatom in the heteroaryl group may be oxidized to N+—(O−), or is benzobthiophenyl;R2 is hydrogen, lower alkyl, halogen, cyano, lower alkyl substituted by hydroxyl, lower alkyl substituted by halogen, lower alkyl substituted by amino, lower alkyl substituted by alkoxy, lower alkyl substituted by amide, or is cycloalkyl;X is CH or N;n is 1 or 2;m is 1 or 2;as well as with a pharmaceutically acceptable salt thereof, with a racemic mixture, or with its corresponding enantiomer and/or optical isomer and/or stereoisomer thereofThe compounds may be used in the treatment of CNS diseases related to positive (psychosis) and negative symptoms of schizophrenia, substance abuse, alcohol and drug addiction, obsessive-compulsive disorders, cognitive impairment, bipolar disorders, mood disorders, major depression, treatment resistant depression, anxiety disorders, Alzheimer's disease, autism, Parkinson's disease, chronic pain, borderline personality disorder, neurodegenerative disease, sleep disturbances, chronic

TREATMENTS FOR AMYOTROPHIC LATERAL SCLEROSIS USING DAZUCORILANT

NºPublicación:  AU2023367284A1 29/05/2025
Solicitante: 
CORCEPT THERAPEUTICS INCORPORATED
CORCEPT THERAPEUTICS INCORPORATED
AU_2023367284_PA

Resumen de: AU2023367284A1

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.

COMPOSITIONS AND METHODS OF TREATING AMYOTROPHIC LATERAL SCLEROSIS (ALS) AND RELATED DISORDERS

NºPublicación:  US2025170212A1 29/05/2025
Solicitante: 
BIOINCEPT LLC [US]
BIOINCEPT, LLC
US_2025170212_PA

Resumen de: US2025170212A1

The present invention relates to methods for the treatment or prevention of symptoms associated with amyotrophic lateral sclerosis (ALS) and related disorders. The present disclosure provides compositions and methods for preventing or treating traumatic brain injuries and other neurological disorders arising from such an injury in a subject by administering to the subject an effective amount of a composition comprising PIF. In certain embodiments, the present disclosure provides compositions and methods for treating traumatic brain injuries and other neurological disorders arising from such an injury in a subject by administering to the subject an effective amount of a composition comprising PIF co-administered with additional agents or treatments.

APPLICATION OF PURIFIED MUSHROOM BETA GLUCAN IN PREVENTING IMPROVING OR TREATING ALZHEIMER'S DISEASE DEMENTIA OR BRAIN FUNCTION DEGENERATION

NºPublicación:  KR20250072854A 26/05/2025
Solicitante: 
시우난첸
KR_20250072854_PA

Resumen de: US2025161387A1

Provided is a method for preventing, improving or treating Alzheimer's disease, dementia or brain function degeneration in a subject in need, including administering to the subject a composition comprising purified mushroom β-glucan, wherein the purified β-glucan is derived from mushroom mycelium or its fermentation product, wherein the purity of the purified mushroom β-glucan is 60% or above.

ROSMARINIC ACID DERIVATIVES AND COMPOSITION CONTAINING THE SAME FOR PREVENTING IMPROVING OR TREATING ALZHEIMER'S DISEASE

NºPublicación:  KR20250071871A 22/05/2025
Solicitante: 
한국과학기술연구원동국대학교산학협력단
KR_20250071871_PA

Resumen de: KR20250071871A

본 발명은 로즈마린산 유도체 및 이를 포함하는 알츠하이머 질환 예방, 개선 또는 치료용 조성물에 관한 것으로, 본 발명에서는 신규한 로즈마린산 유도체 14종을 합성하였으며, 이들 중 아밀로이드-베타(amyloid-β)와 타우 단백질 모두에 대해 플라그 형성을 억제하고 분해를 촉진하는 다중표적 효능을 가진 신물질 2종(RAD-5-9 및 RAD-5-21)을 선별하였다. 따라서, 본 발명의 신규한 로즈마린산 유도체는 알츠하이머 예방, 개선 또는 치료용 조성물로 유용하게 활용할 수 있다.

PEPTIDE CONJUGATE VACCINE COMPOSITIONS AND METHODS FOR THE TREATMENT OF ALZHEIMER'S DISEASE

NºPublicación:  US2025161420A1 22/05/2025
Solicitante: 
MERCK SHARP & DOHME LLC [US]
Merck Sharp & Dohme LLC

Resumen de: US2025161420A1

The invention provides compositions and methods for the treatment of diseases associated with amyloid deposits of Aβ in the brain of a patient, such as Alzheimer's Disease. Such methods entail administering a pharmaceutical composition comprising an immunogenic fragment of Aβ capable of inducing a beneficial immune response in the form of antibodies to Aβ. The immunogenic fragments comprise linear or multivalent peptides of Aβ. Pharmaceutical compositions comprise the immunogenic fragment chemically linked to a carrier molecule which may be administered with an adjuvant.

PEPTIDE CONJUGATE VACCINE COMPOSITIONS AND METHODS FOR THE TREATMENT OF ALZHEIMER'S DISEASE

NºPublicación:  WO2025106603A1 22/05/2025
Solicitante: 
MERCK SHARP & DOHME LLC [US]
MERCK SHARP & DOHME LLC
WO_2025106603_PA

Resumen de: WO2025106603A1

The invention provides compositions and methods for the treatment of diseases associated with amyloid deposits of Aβ in the brain of a patient, such as Alzheimer's Disease. Such methods entail administering a pharmaceutical composition comprising an immunogenic fragment of Aβ capable of inducing a beneficial immune response in the form of antibodies to Aβ. The immunogenic fragments comprise linear or multivalent peptides of Aβ. Pharmaceutical compositions comprise the immunogenic fragment chemically linked to a carrier molecule which may be administered with an adjuvant.

APPLICATION OF PURIFIED MUSHROOM BETA GLUCAN IN PREVENTING, IMPROVING OR TREATING ALZHEIMER'S DISEASE, DEMENTIA OR BRAIN FUNCTION DEGENERATION

NºPublicación:  US2025161387A1 22/05/2025
Solicitante: 
CHEN SHIU NAN [TW]
CHEN Shiu-Nan
CN_120019811_PA

Resumen de: US2025161387A1

Provided is a method for preventing, improving or treating Alzheimer's disease, dementia or brain function degeneration in a subject in need, including administering to the subject a composition comprising purified mushroom β-glucan, wherein the purified β-glucan is derived from mushroom mycelium or its fermentation product, wherein the purity of the purified mushroom β-glucan is 60% or above.

PEPTIDIC INHIBITORS OF AMYLOID SELF- AND CROSS-ASSEMBLY

NºPublicación:  US2025161408A1 22/05/2025
Solicitante: 
TECHNISCHE UNIV MUENCHEN [DE]
LUDWIG MAXIMILIANS UNIV MUENCHEN [DE]
TECHNISCHE UNIVERSIT\u00C4T M\u00DCNCHEN,
LUDWIG-MAXIMILIANS-UNIVERSIT\u00C4T M\u00DCNCHEN
WO_2023161211_A1

Resumen de: US2025161408A1

The present invention relates to a peptide, and to a pharmaceutical composition and a heterocomplex comprising the peptide. Furthermore, the present invention relates to the peptide, the pharmaceutical composition, or the heterocomplex for use in a method of preventing or treating Alzheimer's disease and/or for use in a method of preventing or treating type 2 diabetes. The present invention further relates to the peptide, the pharmaceutical composition, or the heterocomplex for use in a method of diagnosing Alzheimer's disease and/or for use in a method of diagnosing type 2 diabetes. The present invention also relates to a kit for the in vitro or in vivo detection of amyloid fibrils or aggregates, or for the diagnosis of Alzheimer's disease and/or type 2 diabetes in a patient. Moreover, the present invention relates to the use of the peptide or of the heterocomplex in an in vitro assay for the detection of monomeric islet amyloid polypeptide (LAPP), monomeric Aβ40 (42), amyloid fibrils, or amyloid aggregates.

HETEROCYCLIC CARBONYL DERIVATIVE MODULATOR, PREPARATION METHOD THEREFOR, AND USE THEREOF

NºPublicación:  WO2025103475A1 22/05/2025
Solicitante: 
SHANGHAI HANSOH BIOMEDICAL CO LTD [CN]
JIANGSU HANSOH PHARMACEUTICAL GROUP CO LTD [CN]
\u4E0A\u6D77\u7FF0\u68EE\u751F\u7269\u533B\u836F\u79D1\u6280\u6709\u9650\u516C\u53F8,
\u6C5F\u82CF\u8C6A\u68EE\u836F\u4E1A\u96C6\u56E2\u6709\u9650\u516C\u53F8
WO_2025103475_PA

Resumen de: WO2025103475A1

The present invention relates to a heterocyclic carbonyl derivative modulator, a preparation method therefor, and the use thereof. Specifically, the present invention relates to a compound represented by general formula (II-B), a preparation method therefor, a pharmaceutical composition containing said compound, and a use thereof as a modulator in the treatment of Alzheimer's disease, schizophrenia, pain, addiction, and sleep disorders, wherein each substituent in general formula (II-B) is as defined in the description.

USE OF INNATE PHAGOCYTOSIS-PROMOTING POLYPEPTIDE IN PREPARING DRUG FOR TREATING ALZHEIMER'S DISEASE

NºPublicación:  WO2025103285A1 22/05/2025
Solicitante: 
INNATEPHAGO PHARMACEUTICS PTY LTD [CN]
\u957F\u6C99\u82F1\u5948\u53D1\u836F\u4E1A\u6709\u9650\u516C\u53F8
WO_2025103285_PA

Resumen de: WO2025103285A1

Use of an innate phagocytosis-promoting polypeptide in preparing a drug for treating Alzheimer's disease.

IMPROVEMENT OF BLOOD BRAIN BARRIER INTEGRITY

NºPublicación:  WO2025102119A1 22/05/2025
Solicitante: 
CENTENARY INST OF CANCER MEDICINE AND CELL BIOLOGY [AU]
UNIV SYDNEY [AU]
CENTENARY INSTITUTE OF CANCER MEDICINE AND CELL BIOLOGY,
THE UNIVERSITY OF SYDNEY
WO_2025102119_A1

Resumen de: WO2025102119A1

The present disclosure relates generally to the use of agents, such as oligonucleotides, to improve, increase, restore or retain the integrity of the blood brain barrier; and/or to reduce the deposition or accumulation of and/or promote the clearance of, amyloid-β. The disclosure also related to agents, such as oligonucleotides, that increase, promote or restore the level or amount of VE-cadherin in a cell, and are thus useful in the methods of the present disclosure. The present disclosure also relates to the use of such agents to treat diseases and conditions associated with damage to the blood brain barrier, including diseases and conditions associated with neuroinflammation and neurodegeneration, as well as diseases and conditions associated with amyloid-β deposition, including Alzheimer's disease (AD), Cerebral amyloid angiopathy (CAA), Lewy body dementia (LBD) and traumatic brain injury (TBI).

4-AMINOPYRROLO2,1-F1,2,4TRIAZINES AND PREPARATION AND USES THEREOF

NºPublicación:  AU2023371945A1 22/05/2025
Solicitante: 
BIOSPLICE THERAPEUTICS INC
BIOSPLICE THERAPEUTICS, INC
AU_2023371945_A1

Resumen de: AU2023371945A1

4-Aminopyrrolo2,I-f1,2,4triazine compounds of formula I for treating various diseases and pathologies are disclosed. More particularly, the present disclosure concerns the use of 4- aminopyrrolo2,1-fl,2,4triazine compounds or analogs thereof, in the treatment of disorders characterized by overexpression of DYRK1A (e.g., cancer, Down syndrome, Alzheimer's disease, diabetes, and osteoarthritis).

USE OF NOVEL POMALIDOMIDE DERIVATIVE FOR TREATING BRAIN DISEASES

NºPublicación:  US2025161286A1 22/05/2025
Solicitante: 
AEVIS BIO INC [KR]
AEVIS BIO, INC
JP_2025508862_PA

Resumen de: US2025161286A1

The disclosure relates to compositions and methods of using a pomalidomide derivative that exhibits improved pharmacological effects as compared to pomalidomide for treating brain diseases. The pomalidomide derivative exhibits excellent binding to cereblon, has almost no cytotoxicity, is unlikely to cause teratogenic adverse effects, can control TNF-α expression, exhibits excellent in vivo pharmacokinetic stability while suppressing oxidative stress, ameliorates a decrease in motor function which is a symptom of Parkinson's disease, and increases tyrosine hydroxylase expression which is reduced by Parkinson's disease, and thus can be used for preventing or treating brain diseases such as Parkinson's disease.

TREATMENT OF AMYOTROPHIC LATERAL SCLEROSIS

NºPublicación:  US2025163114A1 22/05/2025
Solicitante: 
BRANDEIS UNIV [US]
Brandeis University
US_2023054101_A1

Resumen de: US2025163114A1

Nonsense-mediated mRNA decay (NMD) polypeptides, nucleic acids encoding NMD polypeptides, and methods of using such polypeptides and nucleic acids in the treatment of ALS and in screening for agents for the treatment of ALS are described.

BIFIDOBACTERIUM LONGUM SUBSP. INFANTIS CAPABLE OF RELIEVING PARKINSON'S DISEASE AND USE THEREOF

NºPublicación:  EP4556554A1 21/05/2025
Solicitante: 
WECARE PROBIOTICS CO LTD [CN]
Wecare Probiotics Co., Ltd
EP_4556554_PA

Resumen de: EP4556554A1

Provided are a Bifidobacterium longum subsp. infantis for alleviating Parkinson's disease and a use thereof, and the Bifidobacterium longum subsp. infantis for alleviating Parkinson's disease is named as Bifidobacterium longum subsp. infantis BI03 strain, with a deposit number of CGMCC No.24473 and deposit date of Mar. 7, 2022. The strain can significantly alleviate the symptoms of Parkinson's disease, specifically manifested in: alleviating Parkinson's disease-related dyskinesia and corticosterone elevation; weakening the neuroinflammation associated with Parkinson's disease; promoting glutathione and weakening brain oxidative stress damage.

APPLICATION OF PURIFIED MUSHROOM BETA GLUCAN IN PREVENTING, IMPROVING OR TREATING ALZHEIMER'S DISEASE, DEMENTIA OR BRAIN FUNCTION DEGENERATION

NºPublicación:  EP4556014A1 21/05/2025
Solicitante: 
CHEN SHIU NAN [TW]
Chen, Shiu-Nan
EP_4556014_PA

Resumen de: EP4556014A1

Provided is a method for preventing, improving or treating Alzheimer's disease, dementia or brain function degeneration in a subject in need, including administering to the subject a composition comprising purified mushroom β-glucan, wherein the purified β-glucan is derived from mushroom mycelium or its fermentation product, wherein the purity of the purified mushroom β-glucan is 60% or above.

1 Pharmaceutical composition for treating Parkinson's disease containing cell-transducible Inorganic Pyrophosphatase 1 fusion protein

NºPublicación:  KR20250067974A 16/05/2025
Solicitante: 
한림대학교산학협력단
KR_20250067974_PA

Resumen de: KR20250067974A

과제: 부작용이 적거나 없으며, 효과가 우수한 파킨슨병 예방 또는 치료용 약제를 제공하는 것. 해결수단: 본 발명자들은 PPA1 (Inorganic Pyrophosphatase 1) 단백질에 단백질 수송 도메인을 함께 융합시켜 세포 또는 조직으로의 침투를 가능하게 하여, PPA1 융합단백질이 MPP+와 MPTP로 유도한 도파민성 세포사멸과 파킨슨 동물 질환 모델에서 세포 보호효능을 발현하는지를 연구하였다. 그 결과, 세포 투과성 PPA1 융합단백질은 파킨슨병에서 효과적인 단백질 치료제로서의 가능성이 있음을 확인하였다.

6-Aryl imidazo21-bthi/oxazole-2-carboxilic acid 6-Aryl imidazo21-bthi/oxazole-2-carboxilic acid derivatives and pharmaceutical composition for treating Alzheimer's disease comprising thereof

Nº publicación: KR20250067698A 15/05/2025

Solicitante:

한양대학교에리카산학협력단

KR_20250067698_A

Resumen de: KR20250067698A

본 발명은 6-Aryl imidazo2,1-bthi/oxazole-2-carboxilic acid 유도체에 관한 것으로, 본 발명에 따른 화합물은 JNK3에 대해 특이적으로 작용할 수 있는, JNK3 억제제로 작용한다. 본 발명에 따른 화합물은 JNK1 및 JNK2에 대한 억제 활성에 비하여 JNK3에 대한 억제 활성이 상대적으로 높기 때문에 부작용의 위험성이 낮은 알츠하이머병 치료제로서 활용할 수 있다.

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