LT2588130T - Terapinės priemonės pristatymas cns - Google Patents
Terapinės priemonės pristatymas cnsInfo
- Publication number
- LT2588130T LT2588130T LTEP11799035.8T LT11799035T LT2588130T LT 2588130 T LT2588130 T LT 2588130T LT 11799035 T LT11799035 T LT 11799035T LT 2588130 T LT2588130 T LT 2588130T
- Authority
- LT
- Lithuania
- Prior art keywords
- therapeutic agents
- cns delivery
- cns
- delivery
- therapeutic
- Prior art date
Links
Classifications
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K38/00—Medicinal preparations containing peptides
- A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
- A61K38/43—Enzymes; Proenzymes; Derivatives thereof
- A61K38/46—Hydrolases (3)
- A61K38/465—Hydrolases (3) acting on ester bonds (3.1), e.g. lipases, ribonucleases
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K35/00—Medicinal preparations containing materials or reaction products thereof with undetermined constitution
- A61K35/66—Microorganisms or materials therefrom
- A61K35/76—Viruses; Subviral particles; Bacteriophages
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K35/00—Medicinal preparations containing materials or reaction products thereof with undetermined constitution
- A61K35/66—Microorganisms or materials therefrom
- A61K35/76—Viruses; Subviral particles; Bacteriophages
- A61K35/761—Adenovirus
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K38/00—Medicinal preparations containing peptides
- A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
- A61K38/43—Enzymes; Proenzymes; Derivatives thereof
- A61K38/46—Hydrolases (3)
- A61K38/47—Hydrolases (3) acting on glycosyl compounds (3.2), e.g. cellulases, lactases
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
- A61K47/02—Inorganic compounds
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
- A61K47/06—Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite
- A61K47/26—Carbohydrates, e.g. sugar alcohols, amino sugars, nucleic acids, mono-, di- or oligo-saccharides; Derivatives thereof, e.g. polysorbates, sorbitan fatty acid esters or glycyrrhizin
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K9/00—Medicinal preparations characterised by special physical form
- A61K9/0012—Galenical forms characterised by the site of application
- A61K9/0019—Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K9/00—Medicinal preparations characterised by special physical form
- A61K9/0012—Galenical forms characterised by the site of application
- A61K9/0085—Brain, e.g. brain implants; Spinal cord
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K9/00—Medicinal preparations characterised by special physical form
- A61K9/14—Particulate form, e.g. powders, Processes for size reducing of pure drugs or the resulting products, Pure drug nanoparticles
- A61K9/19—Particulate form, e.g. powders, Processes for size reducing of pure drugs or the resulting products, Pure drug nanoparticles lyophilised, i.e. freeze-dried, solutions or dispersions
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P1/00—Drugs for disorders of the alimentary tract or the digestive system
- A61P1/08—Drugs for disorders of the alimentary tract or the digestive system for nausea, cinetosis or vertigo; Antiemetics
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P1/00—Drugs for disorders of the alimentary tract or the digestive system
- A61P1/16—Drugs for disorders of the alimentary tract or the digestive system for liver or gallbladder disorders, e.g. hepatoprotective agents, cholagogues, litholytics
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P13/00—Drugs for disorders of the urinary system
- A61P13/12—Drugs for disorders of the urinary system of the kidneys
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P21/00—Drugs for disorders of the muscular or neuromuscular system
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P25/00—Drugs for disorders of the nervous system
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P25/00—Drugs for disorders of the nervous system
- A61P25/02—Drugs for disorders of the nervous system for peripheral neuropathies
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P25/00—Drugs for disorders of the nervous system
- A61P25/08—Antiepileptics; Anticonvulsants
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P25/00—Drugs for disorders of the nervous system
- A61P25/14—Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P25/00—Drugs for disorders of the nervous system
- A61P25/28—Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P27/00—Drugs for disorders of the senses
- A61P27/02—Ophthalmic agents
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P27/00—Drugs for disorders of the senses
- A61P27/16—Otologicals
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P3/00—Drugs for disorders of the metabolism
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P9/00—Drugs for disorders of the cardiovascular system
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
- C12N9/14—Hydrolases (3)
- C12N9/24—Hydrolases (3) acting on glycosyl compounds (3.2)
- C12N9/2402—Hydrolases (3) acting on glycosyl compounds (3.2) hydrolysing O- and S- glycosyl compounds (3.2.1)
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12Y—ENZYMES
- C12Y301/00—Hydrolases acting on ester bonds (3.1)
- C12Y301/06—Sulfuric ester hydrolases (3.1.6)
- C12Y301/06013—Iduronate-2-sulfatase (3.1.6.13)
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12Y—ENZYMES
- C12Y302/00—Hydrolases acting on glycosyl compounds, i.e. glycosylases (3.2)
- C12Y302/01—Glycosidases, i.e. enzymes hydrolysing O- and S-glycosyl compounds (3.2.1)
- C12Y302/01045—Glucosylceramidase (3.2.1.45), i.e. beta-glucocerebrosidase
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12Y—ENZYMES
- C12Y302/00—Hydrolases acting on glycosyl compounds, i.e. glycosylases (3.2)
- C12Y302/01—Glycosidases, i.e. enzymes hydrolysing O- and S-glycosyl compounds (3.2.1)
- C12Y302/0105—Alpha-N-acetylglucosaminidase (3.2.1.50)
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K38/00—Medicinal preparations containing peptides
- A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
- A61K38/43—Enzymes; Proenzymes; Derivatives thereof
- A61K38/46—Hydrolases (3)
Applications Claiming Priority (8)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
US35885710P | 2010-06-25 | 2010-06-25 | |
US36078610P | 2010-07-01 | 2010-07-01 | |
US38786210P | 2010-09-29 | 2010-09-29 | |
US201161435710P | 2011-01-24 | 2011-01-24 | |
US201161442115P | 2011-02-11 | 2011-02-11 | |
US201161476210P | 2011-04-15 | 2011-04-15 | |
US201161495268P | 2011-06-09 | 2011-06-09 | |
PCT/US2011/041924 WO2011163648A1 (en) | 2010-06-25 | 2011-06-25 | Cns delivery of therapeutic agents |
Publications (1)
Publication Number | Publication Date |
---|---|
LT2588130T true LT2588130T (lt) | 2016-12-12 |
Family
ID=46888845
Family Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
LTEP11799035.8T LT2588130T (lt) | 2010-06-25 | 2011-06-25 | Terapinės priemonės pristatymas cns |
Country Status (20)
Country | Link |
---|---|
US (1) | US9283181B2 (lt) |
EP (3) | EP3875107A1 (lt) |
JP (1) | JP6045491B2 (lt) |
KR (5) | KR102345973B1 (lt) |
CN (1) | CN103096918B (lt) |
AU (4) | AU2011270668B8 (lt) |
CA (1) | CA2803166C (lt) |
CY (2) | CY1118265T1 (lt) |
DK (1) | DK2588130T3 (lt) |
ES (1) | ES2650689T3 (lt) |
HR (1) | HRP20161505T1 (lt) |
IL (3) | IL291554B2 (lt) |
LT (1) | LT2588130T (lt) |
NZ (1) | NZ605865A (lt) |
PE (1) | PE20230169A1 (lt) |
PL (1) | PL3103469T3 (lt) |
RU (1) | RU2626514C2 (lt) |
UA (1) | UA115648C2 (lt) |
WO (1) | WO2011163648A1 (lt) |
ZA (1) | ZA201300671B (lt) |
Families Citing this family (45)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
JP2006506317A (ja) | 2002-01-11 | 2006-02-23 | バイオマリン ファーマシューティカル インコーポレイテッド | 治療用リソソーム酵素の送達のための酵素送達系としてのp97の使用 |
NZ605865A (en) | 2010-06-25 | 2015-07-31 | Shire Human Genetic Therapies | Cns delivery of therapeutic agents |
CA2803003C (en) | 2010-06-25 | 2022-11-22 | Shire Human Genetic Therapies, Inc. | Methods and compositions for cns delivery of arylsulfatase a |
UA115649C2 (uk) | 2010-06-25 | 2017-12-11 | Шае Хюмен Дженетік Терапіс, Інк. | Способи та композиції для доставки до цнс ідуронат-2-сульфатази |
KR20140005842A (ko) | 2010-06-25 | 2014-01-15 | 샤이어 휴먼 지네틱 테라피즈 인크. | 헤파란 n-설파타제의 cns 전달을 위한 방법들 및 조성물들 |
ES2895655T3 (es) | 2010-06-25 | 2022-02-22 | Shire Human Genetic Therapies | Métodos y composiciones para la administración al SNC de iduronato-2-sulfatasa |
WO2012101671A1 (en) * | 2011-01-25 | 2012-08-02 | Jcr Pharmaceuticals Co., Ltd. | Method for production of human recombinant iduronate 2-sulfatase |
PT2717917T (pt) | 2011-07-05 | 2016-07-27 | Bioasis Technologies Inc | Conjugados de anticorpos p97 |
BR112014008873A2 (pt) * | 2011-10-12 | 2020-09-15 | Synageva Biopharma Corp | proteína naglu recombinante humana e usos da mesma |
EP2793922B1 (en) | 2011-12-23 | 2019-10-30 | Shire Human Genetic Therapies, Inc. | Stable formulations for cns delivery of arylsulfatase a |
BR112014015352A2 (pt) | 2011-12-23 | 2017-06-13 | Shire Human Genetic Therapies | tratamento de comprometimento cognitivo da síndrome de hunter por distribuição intratecal de iduronato-2-sulfatase |
WO2014022515A1 (en) * | 2012-07-31 | 2014-02-06 | Bioasis Technologies, Inc. | Dephosphorylated lysosomal storage disease proteins and methods of use thereof |
EA030551B1 (ru) | 2013-01-09 | 2018-08-31 | Шир Хьюман Дженетик Терапис, Инк. | Способы очистки арилсульфатазы а |
EP2981551B1 (en) | 2013-02-20 | 2020-06-03 | Valerion Therapeutics, LLC | Methods and compositions for treatment of pompe disease |
WO2014160438A1 (en) | 2013-03-13 | 2014-10-02 | Bioasis Technologies Inc. | Fragments of p97 and uses thereof |
CA3209821A1 (en) * | 2013-05-15 | 2014-11-20 | Regents Of The University Of Minnesota | Adeno-associated virus mediated gene transfer to the central nervous system |
CA2935195A1 (en) | 2014-02-03 | 2015-08-06 | Bioasis Technologies, Inc. | P97 fusion proteins |
AU2015219339B2 (en) | 2014-02-19 | 2020-03-05 | Bioasis Technologies Inc. | P97-IDS fusion proteins |
WO2015168521A2 (en) | 2014-05-01 | 2015-11-05 | Bioasis Technologies, Inc. | P97-polynucleotide conjugates |
CA2956469A1 (en) * | 2014-08-11 | 2016-02-18 | Shire Human Genetic Therapies, Inc. | Mannose-6-phosphate bearing peptides fused to lysosomal enzymes |
JP2017531632A (ja) | 2014-09-29 | 2017-10-26 | アレクシオン ファーマシューティカルズ, インコーポレイテッド | ムコ多糖症iiib型(mpsiiib)を治療する方法 |
WO2016100591A1 (en) * | 2014-12-19 | 2016-06-23 | Biogen Ma Inc. | Apparatus for compound dispersion |
AU2016256895B2 (en) | 2015-05-07 | 2022-05-26 | Takeda Pharmaceutical Company Limited | Glucocerebrosidase gene therapy for parkinson's disease |
GB201508025D0 (en) | 2015-05-11 | 2015-06-24 | Ucl Business Plc | Fabry disease gene therapy |
KR20170004814A (ko) * | 2015-07-02 | 2017-01-11 | 주식회사 녹십자 | 헌터증후군 치료제 |
WO2017062666A2 (en) * | 2015-10-06 | 2017-04-13 | Patricia Dickson | Enzyme replacement therapy for mucopolysaccharidosis iiid |
US10123969B2 (en) | 2015-10-15 | 2018-11-13 | Wisconsin Alumni Research Foundation | Osmotic enhancement of drug/therapeutic delivery to the brain following infusion or injection into the cerebrospinal fluid |
SG10201912935WA (en) * | 2015-12-15 | 2020-02-27 | Genzyme Corp | Adeno-associated viral vectors for treating mucolipidosis type ii |
KR20180090387A (ko) * | 2015-12-30 | 2018-08-10 | 주식회사 녹십자 | 헌터증후군을 치료하기 위한 방법 및 조성물 |
EP3416678A1 (en) * | 2016-02-17 | 2018-12-26 | Shire Human Genetic Therapies, Inc. | Methods and compositions for cns delivery of arylsulfatase a |
US11253612B2 (en) | 2016-04-15 | 2022-02-22 | The Trustees Of The University Of Pennsylvania | Gene therapy for treating mucopolysaccharidosis type II |
US11957713B2 (en) | 2016-10-14 | 2024-04-16 | Children's Medical Center Corporation | Compositions and methods for treating diseases and disorders of the central nervous system |
GB201709459D0 (en) | 2017-06-14 | 2017-07-26 | Intrabio Ltd | Treatment for migraine |
WO2019049967A1 (ja) * | 2017-09-07 | 2019-03-14 | Jcrファーマ株式会社 | 水性医薬組成物 |
SG11202002457RA (en) | 2017-09-22 | 2020-04-29 | Univ Pennsylvania | Gene therapy for treating mucopolysaccharidosis type ii |
EP3904389A1 (en) | 2017-10-02 | 2021-11-03 | Denali Therapeutics Inc. | Fusion proteins comprising enzyme replacement therapy enzymes |
CN111542314B (zh) | 2017-10-18 | 2023-11-24 | 内在生物技术有限公司 | 神经退行性疾病的治疗剂 |
US20200338034A1 (en) | 2017-10-18 | 2020-10-29 | Intrabio Ltd | Therapeutic agents for improved mobility and cognitive function and for treating neurodegenerative diseases and lysosomal storage disorders |
JP2021508322A (ja) | 2017-12-19 | 2021-03-04 | シャイアー ヒューマン ジェネティック セラピーズ インコーポレイテッド | 精製されたアリールスルファターゼaおよびその組成物 |
MA51836A (fr) | 2018-02-15 | 2020-12-23 | Intrabio Ltd | Agents thérapeutiques permettant de traiter le syndrome des jambes sans repos |
KR20210024082A (ko) | 2018-06-25 | 2021-03-04 | 제이씨알 파마 가부시키가이샤 | 단백질 함유 수성 액제 |
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US20220362189A1 (en) | 2019-06-28 | 2022-11-17 | Intrabio Ltd. | Combination therapy with acetyl-leucine and miglustat |
WO2021144720A1 (en) | 2020-01-13 | 2021-07-22 | Intrabio Ltd | Treatment of late-onset neurodegenerative diseases in heterozygous npc1 gene mutation carriers |
CA3179105A1 (en) | 2020-05-22 | 2021-11-25 | Michael Strupp | The combination of acetyl leucine and 4-aminopyridine or acetazolamide for treating ataxia |
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JP5570677B2 (ja) * | 2002-04-25 | 2014-08-13 | シャイアー ヒューマン ジェネティック セラピーズ インコーポレイテッド | α−ガラクトシダーゼA欠損症の治療 |
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