HRP20150438T1 - Uporaba lizosomalne kiselinske lipaze za lijeäśenje deficijencije lizosomalne kiselinske lipaze kod pacijenata - Google Patents
Uporaba lizosomalne kiselinske lipaze za lijeäśenje deficijencije lizosomalne kiselinske lipaze kod pacijenata Download PDFInfo
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- HRP20150438T1 HRP20150438T1 HRP20150438TT HRP20150438T HRP20150438T1 HR P20150438 T1 HRP20150438 T1 HR P20150438T1 HR P20150438T T HRP20150438T T HR P20150438TT HR P20150438 T HRP20150438 T HR P20150438T HR P20150438 T1 HRP20150438 T1 HR P20150438T1
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- recombinant human
- lal
- use according
- human lal
- sufficient
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- 108010055297 Sterol Esterase Proteins 0.000 title claims 5
- 208000003221 Lysosomal acid lipase deficiency Diseases 0.000 title claims 3
- 102100026001 Lysosomal acid lipase/cholesteryl ester hydrolase Human genes 0.000 title 1
- 101001004953 Homo sapiens Lysosomal acid lipase/cholesteryl ester hydrolase Proteins 0.000 claims 41
- 210000004185 liver Anatomy 0.000 claims 7
- 108090000340 Transaminases Proteins 0.000 claims 6
- 102000003929 Transaminases Human genes 0.000 claims 6
- 210000002966 serum Anatomy 0.000 claims 6
- 239000003814 drug Substances 0.000 claims 5
- 102000000019 Sterol Esterase Human genes 0.000 claims 4
- GUGOEEXESWIERI-UHFFFAOYSA-N Terfenadine Chemical group C1=CC(C(C)(C)C)=CC=C1C(O)CCCN1CCC(C(O)(C=2C=CC=CC=2)C=2C=CC=CC=2)CC1 GUGOEEXESWIERI-UHFFFAOYSA-N 0.000 claims 4
- 230000001387 anti-histamine Effects 0.000 claims 4
- 239000000739 antihistaminic agent Substances 0.000 claims 4
- 229940124597 therapeutic agent Drugs 0.000 claims 4
- 102100036475 Alanine aminotransferase 1 Human genes 0.000 claims 3
- 108010082126 Alanine transaminase Proteins 0.000 claims 3
- 108010003415 Aspartate Aminotransferases Proteins 0.000 claims 3
- 102000004625 Aspartate Aminotransferases Human genes 0.000 claims 3
- 239000003529 anticholesteremic agent Substances 0.000 claims 3
- 206010067125 Liver injury Diseases 0.000 claims 2
- 230000037396 body weight Effects 0.000 claims 2
- 231100000234 hepatic damage Toxicity 0.000 claims 2
- 102000048394 human LIPA Human genes 0.000 claims 2
- 238000001802 infusion Methods 0.000 claims 2
- 230000008818 liver damage Effects 0.000 claims 2
- NBSCHQHZLSJFNQ-QTVWNMPRSA-N D-Mannose-6-phosphate Chemical compound OC1O[C@H](COP(O)(O)=O)[C@@H](O)[C@H](O)[C@@H]1O NBSCHQHZLSJFNQ-QTVWNMPRSA-N 0.000 claims 1
- WQZGKKKJIJFFOK-QTVWNMPRSA-N D-mannopyranose Chemical compound OC[C@H]1OC(O)[C@@H](O)[C@@H](O)[C@@H]1O WQZGKKKJIJFFOK-QTVWNMPRSA-N 0.000 claims 1
- 102000008857 Ferritin Human genes 0.000 claims 1
- 108050000784 Ferritin Proteins 0.000 claims 1
- 238000008416 Ferritin Methods 0.000 claims 1
- 229940121710 HMGCoA reductase inhibitor Drugs 0.000 claims 1
- 102000001554 Hemoglobins Human genes 0.000 claims 1
- 108010054147 Hemoglobins Proteins 0.000 claims 1
- 206010019842 Hepatomegaly Diseases 0.000 claims 1
- 208000008771 Lymphadenopathy Diseases 0.000 claims 1
- 208000026589 Wolman disease Diseases 0.000 claims 1
- 230000000251 cholesterol ester accumulation Effects 0.000 claims 1
- ZZVUWRFHKOJYTH-UHFFFAOYSA-N diphenhydramine Chemical group C=1C=CC=CC=1C(OCCN(C)C)C1=CC=CC=C1 ZZVUWRFHKOJYTH-UHFFFAOYSA-N 0.000 claims 1
- 229960000520 diphenhydramine Drugs 0.000 claims 1
- 201000010099 disease Diseases 0.000 claims 1
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 claims 1
- OLNTVTPDXPETLC-XPWALMASSA-N ezetimibe Chemical compound N1([C@@H]([C@H](C1=O)CC[C@H](O)C=1C=CC(F)=CC=1)C=1C=CC(O)=CC=1)C1=CC=C(F)C=C1 OLNTVTPDXPETLC-XPWALMASSA-N 0.000 claims 1
- 229960000815 ezetimibe Drugs 0.000 claims 1
- 229940125721 immunosuppressive agent Drugs 0.000 claims 1
- 239000003018 immunosuppressive agent Substances 0.000 claims 1
- 208000018555 lymphatic system disease Diseases 0.000 claims 1
- 238000004519 manufacturing process Methods 0.000 claims 1
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
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K31/00—Medicinal preparations containing organic active ingredients
- A61K31/13—Amines
- A61K31/135—Amines having aromatic rings, e.g. ketamine, nortriptyline
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K31/00—Medicinal preparations containing organic active ingredients
- A61K31/13—Amines
- A61K31/135—Amines having aromatic rings, e.g. ketamine, nortriptyline
- A61K31/138—Aryloxyalkylamines, e.g. propranolol, tamoxifen, phenoxybenzamine
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K31/00—Medicinal preparations containing organic active ingredients
- A61K31/33—Heterocyclic compounds
- A61K31/335—Heterocyclic compounds having oxygen as the only ring hetero atom, e.g. fungichromin
- A61K31/365—Lactones
- A61K31/366—Lactones having six-membered rings, e.g. delta-lactones
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K31/00—Medicinal preparations containing organic active ingredients
- A61K31/33—Heterocyclic compounds
- A61K31/395—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
- A61K31/397—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having four-membered rings, e.g. azetidine
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K45/00—Medicinal preparations containing active ingredients not provided for in groups A61K31/00 - A61K41/00
- A61K45/06—Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
-
- 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
- A61P3/00—Drugs for disorders of the metabolism
- A61P3/06—Antihyperlipidemics
-
- 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)
- C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
- C12N9/18—Carboxylic ester hydrolases (3.1.1)
-
- 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)
- C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
- C12N9/18—Carboxylic ester hydrolases (3.1.1)
- C12N9/20—Triglyceride splitting, e.g. by means of lipase
-
- 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)
- C12Y301/01—Carboxylic ester hydrolases (3.1.1)
- C12Y301/01013—Sterol esterase (3.1.1.13)
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K2300/00—Mixtures or combinations of active ingredients, wherein at least one active ingredient is fully defined in groups A61K31/00 - A61K41/00
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- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- General Health & Medical Sciences (AREA)
- Medicinal Chemistry (AREA)
- Animal Behavior & Ethology (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Pharmacology & Pharmacy (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Engineering & Computer Science (AREA)
- Organic Chemistry (AREA)
- Epidemiology (AREA)
- Genetics & Genomics (AREA)
- Wood Science & Technology (AREA)
- Zoology (AREA)
- Biochemistry (AREA)
- General Engineering & Computer Science (AREA)
- Gastroenterology & Hepatology (AREA)
- Molecular Biology (AREA)
- Biomedical Technology (AREA)
- Biotechnology (AREA)
- Microbiology (AREA)
- Immunology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- General Chemical & Material Sciences (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Hematology (AREA)
- Obesity (AREA)
- Diabetes (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Acyclic And Carbocyclic Compounds In Medicinal Compositions (AREA)
- Enzymes And Modification Thereof (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
- Investigating Or Analysing Biological Materials (AREA)
- Medicines Containing Antibodies Or Antigens For Use As Internal Diagnostic Agents (AREA)
Claims (33)
1. Rekombinantna humana lizosomalna kiselinska lipaza (LAL), naznačena time, da se upotrebljava za liječenje ljudskog pacijenta koji boluje od LAL deficijencije, pomoću njezine primjene na ljudskom pacijentu, gdje je ta primjena dovoljna za normalizaciju razina jetrene transaminaze u serumu kod navedenog ljudskog pacijenta i za smanjenje oštećenja jetre kod navedenog ljudskog pacijenta, te pri čemu se rekombinantna humana LAL primjenjuje na ljudskom pacijentu između jednokratno svakih 7 dana i jednokratno svakih 30 dana.
2. Rekombinantna humana LAL za uporabu prema zahtjevu 1, naznačena time, da je navedena jetrena transaminaza odabrana iz skupine koju čine aspartatna transaminaza seruma (AST) i alaninska transaminaza (ALT).
3. Rekombinantna humana LAL za uporabu prema zahtjevu 2, naznačena time, da navedena jetrena transaminaza je AST.
4. Rekombinantna humana LAL za uporabu prema zahtjevu 2, naznačena time, da navedena jetrena transaminaza je ALT.
5. Rekombinantna humana LAL za uporabu prema zahtjevu 1, naznačena time, da je navedena primjena dovoljna za minimalizaciju hepatomegalije.
6. Rekombinantna humana LAL za uporabu prema zahtjevu 1, naznačena time, da je navedena primjena dovoljna za povećanje razina hemoglobina u serumu.
7. Rekombinantna humana LAL za uporabu prema zahtjevu 1, naznačena time, da je navedena primjena dovoljna za smanjenje veličine jetre.
8. Rekombinantna humana LAL za uporabu prema zahtjevu 1, naznačena time, da je navedena primjena dovoljna za smanjenje razina feritina u serumu.
9. Rekombinantna humana LAL za uporabu prema zahtjevu 1, naznačena time, da se navedena rekombinantna humana LAL primjenjuje jednokratno svakih 7 dana.
10. Rekombinantna humana LAL za uporabu prema zahtjevu 1, naznačena time, da se navedena rekombinantna humana LAL primjenjuje jednokratno svakih 14 dana.
11. Rekombinantna humana LAL za uporabu prema zahtjevu 1, naznačena time, da spomenuti ljudski pacijent pati od Wolmanove bolesti.
12. Rekombinantna humana LAL za uporabu prema zahtjevu 1, naznačena time, da spomenuti ljudski pacijent pati od bolesti nakupljanja kolesterin-estera.
13. Rekombinantna humana LAL za uporabu prema zahtjevu 1, naznačena time, da navedena rekombinantna humana LAL obuhvaća najmanje jednu manozu ili jedan manoza-6-fosfat.
14. Rekombinantna humana LAL za uporabu prema zahtjevu 1, naznačena time, da spomenuta količina rekombinantne humane LAL, koja je dovoljna za normalizaciju razina jetrene transaminaze u serumu, iznosi oko 1 mg po kilogramu tjelesne mase navedenog ljudskog pacijenta.
15. Rekombinantna humana LAL za uporabu prema zahtjevu 1, naznačena time, da spomenuta primjena, koja je dovoljna za postizanje polu-života (t1/2) za LAL, traje manje od 20 minuta.
16. Rekombinantna humana LAL za uporabu prema zahtjevu 1, naznačena time, da spomenuta primjena, koja je dovoljna za postizanje polu-života (t1/2) za LAL, traje oko 6, 7, 8, 9, 10, 11, 12, 13, 14, 15, 16 ili 17 minuta.
17. Rekombinantna humana LAL za uporabu prema zahtjevu 1, naznačena time, da je spomenuta primjena dovoljna za postizanje Cmax od 200 ng/mL do 1500 ng/mL.
18. Rekombinantna humana LAL za uporabu prema zahtjevu 1, naznačena time, da je spomenuta primjena dovoljna za postizanje Cmax od 200 ng/mL do 800 ng/mL.
19. Rekombinantna humana LAL za uporabu prema zahtjevu 1, naznačena time, da se spomenuta rekombinantna humana LAL primjenjuje intravenozno.
20. Rekombinantna humana LAL za uporabu prema zahtjevu 19, naznačena time, da je spomenuta primjena pomoću infuzije.
21. Rekombinantna humana LAL za uporabu prema zahtjevu 20, naznačena time, da je spomenuta infuzija za vrijeme od jednog do četiri sata.
22. Rekombinantna humana LAL za uporabu prema zahtjevu 1, naznačena time, da je spomenuta primjena dovoljna za smanjenje limfadenopatije.
23. Rekombinantna humana LAL za uporabu prema zahtjevu 1, naznačena time, da je navedeni ljudski pacijent mlađi od 1 godine života i spomenuta primjena je dovoljna za povećanje brzine rasta navedenog ljudskog pacijenta.
24. Rekombinantna humana LAL za uporabu prema zahtjevu 1, naznačena time, da nadalje obuhvaća primjenu drugog terapeutskog sredstva.
25. Rekombinantna humana LAL za uporabu prema zahtjevu 24, naznačena time, da navedeno drugo terapeutsko sredstvo je lijek za smanjenje kolesterola.
26. Rekombinantna humana LAL za uporabu prema zahtjevu 25, naznačena time, da navedeni lijek za smanjenje kolesterola je statin.
27. Rekombinantna humana LAL za uporabu prema zahtjevu 25, naznačena time, da navedeni lijek za smanjenje kolesterola je ezetimib.
28. Rekombinantna humana LAL za uporabu prema zahtjevu 24, naznačena time, da navedeno drugo terapeutsko sredstvo je imunosupresivno sredstvo.
29. Rekombinantna humana LAL za uporabu prema zahtjevu 24, naznačena time, da navedeno drugo terapeutsko sredstvo je antihistamin.
30. Rekombinantna humana LAL za uporabu prema zahtjevu 29, naznačena time, da navedeni antihistamin je difenhidramin.
31. Rekombinantna humana LAL za uporabu prema zahtjevu 29 ili 30, naznačena time, da se antihistamin primjenjuje u dozi od 1 mg do 5 mg po kilogramu tjelesne mase ljudskog pacijenta.
32. Rekombinantna humana LAL za uporabu prema zahtjevu 29 ili 30, naznačena time, da se antihistamin primjenjuje između 20 i 90 minuta prije primjene rekombinantne humane LAL.
33. Uporaba rekombinantne humane lizosomalne kiselinske lipaze (LAL), naznačena time, da je za proizvodnju lijeka za liječenje ljudskog pacijenta koji boluje od LAL deficijencije, pomoću normalizacije razina jetrene transaminaze u serumu od navedenog ljudskog pacijenta te za smanjenje oštećenja jetre kod navedenog ljudskog pacijenta, pri čemu se rekombinantna humana LAL primjenjuje na ljudskom pacijentu između jednokratno svakih 7 dana i jednokratno svakih 30 dana, kako je definirano u bilo kojem od zahtjeva 1 do 32.
Applications Claiming Priority (6)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US40301110P | 2010-09-09 | 2010-09-09 | |
| US45601410P | 2010-10-29 | 2010-10-29 | |
| US201161432372P | 2011-01-13 | 2011-01-13 | |
| PCT/US2011/033699 WO2011133960A2 (en) | 2010-04-23 | 2011-04-23 | Lysosomal storage disease enzyme |
| EP11758644.6A EP2613798B2 (en) | 2010-09-09 | 2011-09-09 | Use of lysosomal acid lipase for treating lysosomal acid lipase deficiency in patients |
| PCT/US2011/051096 WO2012050695A1 (en) | 2010-09-09 | 2011-09-09 | Use of lysosomal acid lipase for treating lysosomal acid lipase deficiency in patients |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| HRP20150438T1 true HRP20150438T1 (hr) | 2015-05-22 |
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| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| HRP20150438TT HRP20150438T1 (hr) | 2010-09-09 | 2015-04-21 | Uporaba lizosomalne kiselinske lipaze za lijeäśenje deficijencije lizosomalne kiselinske lipaze kod pacijenata |
Country Status (30)
| Country | Link |
|---|---|
| US (5) | US8663631B2 (hr) |
| EP (3) | EP2977057B1 (hr) |
| JP (3) | JP5693728B2 (hr) |
| KR (1) | KR20150038636A (hr) |
| CN (2) | CN103200958A (hr) |
| AR (1) | AR082953A1 (hr) |
| AU (1) | AU2011314293B2 (hr) |
| BR (2) | BR112013005673B1 (hr) |
| CA (2) | CA3209456A1 (hr) |
| CL (1) | CL2013000664A1 (hr) |
| CO (1) | CO6710913A2 (hr) |
| CY (1) | CY1116544T1 (hr) |
| DK (2) | DK2977057T3 (hr) |
| ES (2) | ES2769836T3 (hr) |
| HK (2) | HK1220907A1 (hr) |
| HR (1) | HRP20150438T1 (hr) |
| HU (1) | HUE048688T2 (hr) |
| IL (2) | IL225095A (hr) |
| ME (1) | ME02062B (hr) |
| MX (2) | MX2013002704A (hr) |
| NZ (3) | NZ700824A (hr) |
| PL (2) | PL2977057T3 (hr) |
| PT (2) | PT2977057T (hr) |
| RS (1) | RS53947B1 (hr) |
| RU (1) | RU2550961C2 (hr) |
| SG (2) | SG188456A1 (hr) |
| SI (2) | SI2977057T1 (hr) |
| SM (1) | SMT201500108B (hr) |
| TW (1) | TWI482625B (hr) |
| WO (1) | WO2012050695A1 (hr) |
Families Citing this family (13)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| EP1267914B2 (en) | 2000-02-04 | 2013-11-06 | Children's Hospital Research Foundation | Use of lysosomal acid lipase for treating atherosclerosis and related diseases |
| US20090297496A1 (en) * | 2005-09-08 | 2009-12-03 | Childrens Hospital Medical Center | Lysosomal Acid Lipase Therapy for NAFLD and Related Diseases |
| AU2011242461B2 (en) | 2010-04-23 | 2015-10-01 | Alexion Pharmaceuticals, Inc. | Lysosomal storage disease enzyme |
| CA3209456A1 (en) | 2010-09-09 | 2012-04-19 | Alexion Pharmaceuticals, Inc. | Use of lysosomal acid lipase for treating lysosomal acid lipase deficiency in patients |
| WO2012112681A1 (en) | 2011-02-15 | 2012-08-23 | Shire Human Genetic Therapies, Inc. | Methods for treating lysosomal acid lipase deficiency |
| KR20140108519A (ko) | 2011-10-12 | 2014-09-11 | 시나게바 바이오파르마, 코포레이션 | 재조합 인간 naglu 단백질 및 이의 용도 |
| WO2013131014A1 (en) * | 2012-03-02 | 2013-09-06 | Synageva Biopharma Corp. | Truncated lysosomal acid lipase |
| CN106885910B (zh) * | 2015-12-15 | 2018-12-21 | 上海吉涛生物科技有限公司 | 肿瘤易感微环境检测装置及试剂盒 |
| US11235036B2 (en) | 2016-06-17 | 2022-02-01 | Alexion Pharmaceuticals, Inc. | Lysosomal acid lipase deficiency compositions and methods |
| US11155794B2 (en) | 2016-08-23 | 2021-10-26 | Alexion Pharmaceuticals, Inc. | Method of purifying a heterologous protein from an egg white |
| US10633705B2 (en) | 2016-10-14 | 2020-04-28 | Alexion Pharmaceuticals, Inc. | N-acetyl-alpha-D-glucosaminidase deficiency compositions and methods |
| US11396499B2 (en) | 2018-12-12 | 2022-07-26 | University Of Washington | Lysosomal acid lipase assay |
| WO2021117122A1 (ja) * | 2019-12-10 | 2021-06-17 | 株式会社リボルナバイオサイエンス | ライソゾーム酸性リパーゼ欠損症の予防または治療剤 |
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| SE9001985D0 (sv) | 1990-06-01 | 1990-06-01 | Astra Ab | New chemical products |
| WO1992016212A1 (en) | 1991-03-13 | 1992-10-01 | The United States Of America, As Represented By The Secretary, U.S. Department Of Commerce | Increasing the therapeutic efficiency of macrophage-targeted therapeutic agents by up-regulating the mannose lectin on macrophages |
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