JP2016040335A5 - - Google Patents
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- JP2016040335A5 JP2016040335A5 JP2015246258A JP2015246258A JP2016040335A5 JP 2016040335 A5 JP2016040335 A5 JP 2016040335A5 JP 2015246258 A JP2015246258 A JP 2015246258A JP 2015246258 A JP2015246258 A JP 2015246258A JP 2016040335 A5 JP2016040335 A5 JP 2016040335A5
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- 238000009472 formulation Methods 0.000 claims description 56
- 239000000203 mixture Substances 0.000 claims description 56
- FAPWRFPIFSIZLT-UHFFFAOYSA-M Sodium chloride Chemical group [Na+].[Cl-] FAPWRFPIFSIZLT-UHFFFAOYSA-M 0.000 claims description 14
- 239000011780 sodium chloride Substances 0.000 claims description 7
- 229920001213 Polysorbate 20 Polymers 0.000 claims description 4
- 239000000256 polyoxyethylene sorbitan monolaurate Substances 0.000 claims description 4
- 235000010486 polyoxyethylene sorbitan monolaurate Nutrition 0.000 claims description 4
- 229940068977 polysorbate 20 Drugs 0.000 claims description 4
- 108010089932 heparan sulfate sulfatase Proteins 0.000 claims 18
- 238000007913 intrathecal administration Methods 0.000 claims 14
- 210000001519 tissue Anatomy 0.000 claims 10
- 230000000694 effects Effects 0.000 claims 6
- 229920000136 polysorbate Polymers 0.000 claims 6
- 229950008882 polysorbate Drugs 0.000 claims 6
- 239000004094 surface-active agent Substances 0.000 claims 6
- 229910019142 PO4 Inorganic materials 0.000 claims 5
- 230000002093 peripheral effect Effects 0.000 claims 5
- NBIIXXVUZAFLBC-UHFFFAOYSA-K phosphate Chemical compound [O-]P([O-])([O-])=O NBIIXXVUZAFLBC-UHFFFAOYSA-K 0.000 claims 5
- 239000010452 phosphate Substances 0.000 claims 5
- 229930006000 Sucrose Natural products 0.000 claims 4
- 239000003381 stabilizer Substances 0.000 claims 4
- 239000005720 sucrose Substances 0.000 claims 4
- WQZGKKKJIJFFOK-GASJEMHNSA-N Glucose Natural products OC[C@H]1OC(O)[C@H](O)[C@@H](O)[C@@H]1O WQZGKKKJIJFFOK-GASJEMHNSA-N 0.000 claims 3
- 210000004556 brain Anatomy 0.000 claims 3
- 239000008103 glucose Substances 0.000 claims 3
- 238000001990 intravenous administration Methods 0.000 claims 3
- 230000002132 lysosomal effect Effects 0.000 claims 3
- 210000002569 neuron Anatomy 0.000 claims 3
- 108090000623 proteins and genes Proteins 0.000 claims 3
- 102000004169 proteins and genes Human genes 0.000 claims 3
- 150000003839 salts Chemical class 0.000 claims 3
- 210000005250 spinal neuron Anatomy 0.000 claims 3
- 208000011580 syndromic disease Diseases 0.000 claims 3
- CZMRCDWAGMRECN-UGDNZRGBSA-N Sucrose Chemical compound O[C@H]1[C@H](O)[C@@H](CO)O[C@@]1(CO)O[C@@H]1[C@H](O)[C@@H](O)[C@H](O)[C@@H](CO)O1 CZMRCDWAGMRECN-UGDNZRGBSA-N 0.000 claims 2
- 238000009825 accumulation Methods 0.000 claims 2
- 210000005013 brain tissue Anatomy 0.000 claims 2
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 claims 2
- 210000004705 lumbosacral region Anatomy 0.000 claims 2
- 238000000034 method Methods 0.000 claims 2
- 239000000843 powder Substances 0.000 claims 2
- 125000000185 sucrose group Chemical group 0.000 claims 2
- 208000024891 symptom Diseases 0.000 claims 2
- 239000004475 Arginine Substances 0.000 claims 1
- FBPFZTCFMRRESA-FSIIMWSLSA-N D-Glucitol Natural products OC[C@H](O)[C@H](O)[C@@H](O)[C@H](O)CO FBPFZTCFMRRESA-FSIIMWSLSA-N 0.000 claims 1
- FBPFZTCFMRRESA-KVTDHHQDSA-N D-Mannitol Chemical compound OC[C@@H](O)[C@@H](O)[C@H](O)[C@H](O)CO FBPFZTCFMRRESA-KVTDHHQDSA-N 0.000 claims 1
- FBPFZTCFMRRESA-JGWLITMVSA-N D-glucitol Chemical compound OC[C@H](O)[C@@H](O)[C@H](O)[C@H](O)CO FBPFZTCFMRRESA-JGWLITMVSA-N 0.000 claims 1
- 206010011878 Deafness Diseases 0.000 claims 1
- 208000012239 Developmental disease Diseases 0.000 claims 1
- 229920002971 Heparan sulfate Polymers 0.000 claims 1
- ODKSFYDXXFIFQN-BYPYZUCNSA-P L-argininium(2+) Chemical compound NC(=[NH2+])NCCC[C@H]([NH3+])C(O)=O ODKSFYDXXFIFQN-BYPYZUCNSA-P 0.000 claims 1
- HNDVDQJCIGZPNO-YFKPBYRVSA-N L-histidine Chemical compound OC(=O)[C@@H](N)CC1=CN=CN1 HNDVDQJCIGZPNO-YFKPBYRVSA-N 0.000 claims 1
- KDXKERNSBIXSRK-YFKPBYRVSA-N L-lysine Chemical compound NCCCC[C@H](N)C(O)=O KDXKERNSBIXSRK-YFKPBYRVSA-N 0.000 claims 1
- KDXKERNSBIXSRK-UHFFFAOYSA-N Lysine Natural products NCCCCC(N)C(O)=O KDXKERNSBIXSRK-UHFFFAOYSA-N 0.000 claims 1
- 239000004472 Lysine Substances 0.000 claims 1
- 229930195725 Mannitol Natural products 0.000 claims 1
- 208000036626 Mental retardation Diseases 0.000 claims 1
- 229920001030 Polyethylene Glycol 4000 Polymers 0.000 claims 1
- 229920001219 Polysorbate 40 Polymers 0.000 claims 1
- 229920001214 Polysorbate 60 Polymers 0.000 claims 1
- 210000001744 T-lymphocyte Anatomy 0.000 claims 1
- 230000003044 adaptive effect Effects 0.000 claims 1
- 230000002411 adverse Effects 0.000 claims 1
- 230000016571 aggressive behavior Effects 0.000 claims 1
- 150000001413 amino acids Chemical group 0.000 claims 1
- 239000003708 ampul Substances 0.000 claims 1
- 210000000576 arachnoid Anatomy 0.000 claims 1
- ODKSFYDXXFIFQN-UHFFFAOYSA-N arginine Natural products OC(=O)C(N)CCCNC(N)=N ODKSFYDXXFIFQN-UHFFFAOYSA-N 0.000 claims 1
- WQZGKKKJIJFFOK-VFUOTHLCSA-N beta-D-glucose Chemical compound OC[C@H]1O[C@@H](O)[C@H](O)[C@@H](O)[C@@H]1O WQZGKKKJIJFFOK-VFUOTHLCSA-N 0.000 claims 1
- 210000004027 cell Anatomy 0.000 claims 1
- 210000001638 cerebellum Anatomy 0.000 claims 1
- 210000003710 cerebral cortex Anatomy 0.000 claims 1
- 230000003111 delayed effect Effects 0.000 claims 1
- 201000010099 disease Diseases 0.000 claims 1
- 208000035475 disorder Diseases 0.000 claims 1
- 239000002552 dosage form Substances 0.000 claims 1
- 239000003814 drug Substances 0.000 claims 1
- 229940079593 drug Drugs 0.000 claims 1
- 238000002651 drug therapy Methods 0.000 claims 1
- 125000002791 glucosyl group Chemical group C1([C@H](O)[C@@H](O)[C@H](O)[C@H](O1)CO)* 0.000 claims 1
- 210000004884 grey matter Anatomy 0.000 claims 1
- 230000010370 hearing loss Effects 0.000 claims 1
- 231100000888 hearing loss Toxicity 0.000 claims 1
- 208000016354 hearing loss disease Diseases 0.000 claims 1
- HNDVDQJCIGZPNO-UHFFFAOYSA-N histidine Natural products OC(=O)C(N)CC1=CN=CN1 HNDVDQJCIGZPNO-UHFFFAOYSA-N 0.000 claims 1
- 208000013403 hyperactivity Diseases 0.000 claims 1
- 239000003018 immunosuppressive agent Substances 0.000 claims 1
- 229940124589 immunosuppressive drug Drugs 0.000 claims 1
- 210000003734 kidney Anatomy 0.000 claims 1
- 230000021633 leukocyte mediated immunity Effects 0.000 claims 1
- 239000012669 liquid formulation Substances 0.000 claims 1
- 210000004185 liver Anatomy 0.000 claims 1
- 230000004807 localization Effects 0.000 claims 1
- 239000000594 mannitol Substances 0.000 claims 1
- 235000010355 mannitol Nutrition 0.000 claims 1
- 239000012528 membrane Substances 0.000 claims 1
- 210000004379 membrane Anatomy 0.000 claims 1
- 210000002418 meninge Anatomy 0.000 claims 1
- 210000001259 mesencephalon Anatomy 0.000 claims 1
- 239000002052 molecular layer Substances 0.000 claims 1
- 210000000478 neocortex Anatomy 0.000 claims 1
- 230000001537 neural effect Effects 0.000 claims 1
- 210000004498 neuroglial cell Anatomy 0.000 claims 1
- 210000004786 perivascular cell Anatomy 0.000 claims 1
- 150000003904 phospholipids Chemical class 0.000 claims 1
- 239000000244 polyoxyethylene sorbitan monooleate Substances 0.000 claims 1
- 235000010482 polyoxyethylene sorbitan monooleate Nutrition 0.000 claims 1
- 239000000249 polyoxyethylene sorbitan monopalmitate Substances 0.000 claims 1
- 235000010483 polyoxyethylene sorbitan monopalmitate Nutrition 0.000 claims 1
- 239000001818 polyoxyethylene sorbitan monostearate Substances 0.000 claims 1
- 235000010989 polyoxyethylene sorbitan monostearate Nutrition 0.000 claims 1
- 229940101027 polysorbate 40 Drugs 0.000 claims 1
- 229940113124 polysorbate 60 Drugs 0.000 claims 1
- 229920000053 polysorbate 80 Polymers 0.000 claims 1
- 229940068968 polysorbate 80 Drugs 0.000 claims 1
- 229940071643 prefilled syringe Drugs 0.000 claims 1
- 210000000449 purkinje cell Anatomy 0.000 claims 1
- 208000019116 sleep disease Diseases 0.000 claims 1
- 208000020685 sleep-wake disease Diseases 0.000 claims 1
- 239000000600 sorbitol Substances 0.000 claims 1
- 210000000278 spinal cord Anatomy 0.000 claims 1
- 238000012385 systemic delivery Methods 0.000 claims 1
- 238000012384 transportation and delivery Methods 0.000 claims 1
- 230000002477 vacuolizing effect Effects 0.000 claims 1
- 210000004885 white matter Anatomy 0.000 claims 1
- LOKCTEFSRHRXRJ-UHFFFAOYSA-I dipotassium trisodium dihydrogen phosphate hydrogen phosphate dichloride Chemical compound P(=O)(O)(O)[O-].[K+].P(=O)(O)([O-])[O-].[Na+].[Na+].[Cl-].[K+].[Cl-].[Na+] LOKCTEFSRHRXRJ-UHFFFAOYSA-I 0.000 description 3
- 239000002953 phosphate buffered saline Substances 0.000 description 3
- 239000002904 solvent Substances 0.000 description 2
- 238000001802 infusion Methods 0.000 description 1
- 239000001488 sodium phosphate Substances 0.000 description 1
- 229910000162 sodium phosphate Inorganic materials 0.000 description 1
- RYFMWSXOAZQYPI-UHFFFAOYSA-K trisodium phosphate Chemical compound [Na+].[Na+].[Na+].[O-]P([O-])([O-])=O RYFMWSXOAZQYPI-UHFFFAOYSA-K 0.000 description 1
Applications Claiming Priority (14)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US35885710P | 2010-06-25 | 2010-06-25 | |
| US61/358,857 | 2010-06-25 | ||
| US36078610P | 2010-07-01 | 2010-07-01 | |
| US61/360,786 | 2010-07-01 | ||
| US38786210P | 2010-09-29 | 2010-09-29 | |
| US61/387,862 | 2010-09-29 | ||
| US201161435710P | 2011-01-24 | 2011-01-24 | |
| US61/435,710 | 2011-01-24 | ||
| US201161442115P | 2011-02-11 | 2011-02-11 | |
| US61/442,115 | 2011-02-11 | ||
| US201161476210P | 2011-04-15 | 2011-04-15 | |
| US61/476,210 | 2011-04-15 | ||
| US201161495268P | 2011-06-09 | 2011-06-09 | |
| US61/495,268 | 2011-06-09 |
Related Parent Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2013516843A Division JP6073783B2 (ja) | 2010-06-25 | 2011-06-25 | ヘパランｎ−スルファターゼのｃｎｓ送達のための方法および組成物 |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2016040335A JP2016040335A (ja) | 2016-03-24 |
| JP2016040335A5 true JP2016040335A5 (OSRAM) | 2017-04-20 |
Family
ID=45352777
Family Applications (22)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2015236539A Active JP6250616B2 (ja) | 2010-06-25 | 2015-12-03 | 治療薬のｃｎｓ送達 |
| JP2015246258A Withdrawn JP2016040335A (ja) | 2010-06-25 | 2015-12-17 | ヘパランｎ−スルファターゼのｃｎｓ送達のための方法および組成物 |
| JP2015246244A Active JP6285409B2 (ja) | 2010-06-25 | 2015-12-17 | イズロン酸−２−スルファターゼのｃｎｓ送達のための方法および組成物 |
| JP2015246589A Pending JP2016094451A (ja) | 2010-06-25 | 2015-12-17 | サンフィリポ症候群ｂ型の処置 |
| JP2015246952A Active JP6346162B2 (ja) | 2010-06-25 | 2015-12-18 | アリールスルファターゼａのｃｎｓ送達の方法および組成物 |
| JP2017177784A Active JP6466538B2 (ja) | 2010-06-25 | 2017-09-15 | 治療薬のｃｎｓ送達 |
| JP2017194348A Active JP6522072B2 (ja) | 2010-06-25 | 2017-10-04 | イズロン酸−２−スルファターゼのｃｎｓ送達のための方法および組成物 |
| JP2017194508A Active JP6522073B2 (ja) | 2010-06-25 | 2017-10-04 | アリールスルファターゼａのｃｎｓ送達の方法および組成物 |
| JP2018192589A Active JP6797876B2 (ja) | 2010-06-25 | 2018-10-11 | 治療薬のｃｎｓ送達 |
| JP2018226358A Active JP6898909B2 (ja) | 2010-06-25 | 2018-12-03 | アリールスルファターゼａのｃｎｓ送達の方法および組成物 |
| JP2018227369A Active JP6938456B2 (ja) | 2010-06-25 | 2018-12-04 | イズロン酸−２−スルファターゼのｃｎｓ送達のための方法および組成物 |
| JP2020000930A Withdrawn JP2020079244A (ja) | 2010-06-25 | 2020-01-07 | 治療薬のｃｎｓ送達 |
| JP2020023620A Pending JP2020079297A (ja) | 2010-06-25 | 2020-02-14 | アリールスルファターゼａのｃｎｓ送達の方法および組成物 |
| JP2020024183A Pending JP2020079298A (ja) | 2010-06-25 | 2020-02-17 | イズロン酸−２−スルファターゼのｃｎｓ送達のための方法および組成物 |
| JP2021115564A Active JP7087175B2 (ja) | 2010-06-25 | 2021-07-13 | 治療薬のｃｎｓ送達 |
| JP2021115560A Active JP7448507B2 (ja) | 2010-06-25 | 2021-07-13 | アリールスルファターゼａのｃｎｓ送達の方法および組成物 |
| JP2021133184A Active JP7591476B2 (ja) | 2010-06-25 | 2021-08-18 | イズロン酸－２－スルファターゼのｃｎｓ送達のための方法および組成物 |
| JP2021196898A Active JP7359827B2 (ja) | 2010-06-25 | 2021-12-03 | 治療薬のｃｎｓ送達 |
| JP2023014814A Active JP7543459B2 (ja) | 2010-06-25 | 2023-02-02 | アリールスルファターゼａのｃｎｓ送達の方法および組成物 |
| JP2023024170A Pending JP2023062114A (ja) | 2010-06-25 | 2023-02-20 | イズロン酸－２－スルファターゼのｃｎｓ送達のための方法および組成物 |
| JP2023078673A Pending JP2023099216A (ja) | 2010-06-25 | 2023-05-11 | 治療薬のｃｎｓ送達 |
| JP2024069806A Pending JP2024097029A (ja) | 2010-06-25 | 2024-04-23 | アリールスルファターゼａのｃｎｓ送達の方法および組成物 |
Family Applications Before (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2015236539A Active JP6250616B2 (ja) | 2010-06-25 | 2015-12-03 | 治療薬のｃｎｓ送達 |
Family Applications After (20)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2015246244A Active JP6285409B2 (ja) | 2010-06-25 | 2015-12-17 | イズロン酸−２−スルファターゼのｃｎｓ送達のための方法および組成物 |
| JP2015246589A Pending JP2016094451A (ja) | 2010-06-25 | 2015-12-17 | サンフィリポ症候群ｂ型の処置 |
| JP2015246952A Active JP6346162B2 (ja) | 2010-06-25 | 2015-12-18 | アリールスルファターゼａのｃｎｓ送達の方法および組成物 |
| JP2017177784A Active JP6466538B2 (ja) | 2010-06-25 | 2017-09-15 | 治療薬のｃｎｓ送達 |
| JP2017194348A Active JP6522072B2 (ja) | 2010-06-25 | 2017-10-04 | イズロン酸−２−スルファターゼのｃｎｓ送達のための方法および組成物 |
| JP2017194508A Active JP6522073B2 (ja) | 2010-06-25 | 2017-10-04 | アリールスルファターゼａのｃｎｓ送達の方法および組成物 |
| JP2018192589A Active JP6797876B2 (ja) | 2010-06-25 | 2018-10-11 | 治療薬のｃｎｓ送達 |
| JP2018226358A Active JP6898909B2 (ja) | 2010-06-25 | 2018-12-03 | アリールスルファターゼａのｃｎｓ送達の方法および組成物 |
| JP2018227369A Active JP6938456B2 (ja) | 2010-06-25 | 2018-12-04 | イズロン酸−２−スルファターゼのｃｎｓ送達のための方法および組成物 |
| JP2020000930A Withdrawn JP2020079244A (ja) | 2010-06-25 | 2020-01-07 | 治療薬のｃｎｓ送達 |
| JP2020023620A Pending JP2020079297A (ja) | 2010-06-25 | 2020-02-14 | アリールスルファターゼａのｃｎｓ送達の方法および組成物 |
| JP2020024183A Pending JP2020079298A (ja) | 2010-06-25 | 2020-02-17 | イズロン酸−２−スルファターゼのｃｎｓ送達のための方法および組成物 |
| JP2021115564A Active JP7087175B2 (ja) | 2010-06-25 | 2021-07-13 | 治療薬のｃｎｓ送達 |
| JP2021115560A Active JP7448507B2 (ja) | 2010-06-25 | 2021-07-13 | アリールスルファターゼａのｃｎｓ送達の方法および組成物 |
| JP2021133184A Active JP7591476B2 (ja) | 2010-06-25 | 2021-08-18 | イズロン酸－２－スルファターゼのｃｎｓ送達のための方法および組成物 |
| JP2021196898A Active JP7359827B2 (ja) | 2010-06-25 | 2021-12-03 | 治療薬のｃｎｓ送達 |
| JP2023014814A Active JP7543459B2 (ja) | 2010-06-25 | 2023-02-02 | アリールスルファターゼａのｃｎｓ送達の方法および組成物 |
| JP2023024170A Pending JP2023062114A (ja) | 2010-06-25 | 2023-02-20 | イズロン酸－２－スルファターゼのｃｎｓ送達のための方法および組成物 |
| JP2023078673A Pending JP2023099216A (ja) | 2010-06-25 | 2023-05-11 | 治療薬のｃｎｓ送達 |
| JP2024069806A Pending JP2024097029A (ja) | 2010-06-25 | 2024-04-23 | アリールスルファターゼａのｃｎｓ送達の方法および組成物 |
Country Status (24)
| Country | Link |
|---|---|
| US (17) | US9814764B2 (OSRAM) |
| JP (22) | JP6250616B2 (OSRAM) |
| CN (4) | CN105664142A (OSRAM) |
| AR (6) | AR081680A1 (OSRAM) |
| BR (1) | BR112012033214B1 (OSRAM) |
| CA (1) | CA3171308A1 (OSRAM) |
| CL (7) | CL2012003656A1 (OSRAM) |
| DK (3) | DK3626258T3 (OSRAM) |
| ES (3) | ES2895655T3 (OSRAM) |
| HK (1) | HK1214149A1 (OSRAM) |
| HR (3) | HRP20211660T1 (OSRAM) |
| HU (6) | HUE052944T2 (OSRAM) |
| LT (3) | LT3626257T (OSRAM) |
| MX (1) | MX354776B (OSRAM) |
| NZ (2) | NZ702800A (OSRAM) |
| PE (8) | PE20130637A1 (OSRAM) |
| PL (1) | PL2588130T3 (OSRAM) |
| PT (6) | PT2593131T (OSRAM) |
| RS (3) | RS61683B1 (OSRAM) |
| RU (1) | RU2761342C2 (OSRAM) |
| SI (3) | SI3626257T1 (OSRAM) |
| SM (1) | SMT201600385B (OSRAM) |
| TW (9) | TWI698252B (OSRAM) |
| UA (3) | UA125743C2 (OSRAM) |
Families Citing this family (30)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2008022349A2 (en) | 2006-08-18 | 2008-02-21 | Armagen Technologies, Inc. | Agents for blood-brain barrier delivery |
| EP2997976A1 (en) | 2007-07-27 | 2016-03-23 | Armagen Technologies, Inc. | Methods and compositions for increasing alpha-l-iduronidase activity in the cns |
| EP2485761B1 (en) | 2009-10-09 | 2019-02-27 | Armagen, Inc. | Methods and compositions for increasing iduronate 2-sulfatase activity in the cns |
| LT2588130T (lt) | 2010-06-25 | 2016-12-12 | Shire Human Genetic Therapies, Inc. | Terapinės priemonės pristatymas cns |
| US20220133863A1 (en) * | 2010-06-25 | 2022-05-05 | Shire Human Genetic Therapies, Inc. | Treatment of sanfilippo syndrome type b |
| MX344795B (es) | 2010-06-25 | 2017-01-05 | Shire Human Genetic Therapies | Composiciones y metodos para suministro al sistema nervioso central de arilsulfatasa a. |
| BR112012033197A2 (pt) | 2010-06-25 | 2017-06-06 | Shire Human Genetic Therapies | métodos e composições para liberação de cns de n-sulfatase de heparano. |
| RS61683B1 (sr) | 2010-06-25 | 2021-05-31 | Shire Human Genetic Therapies | Isporuka terapijskih agenasa u centralni nervni sistem |
| EP2785378B1 (en) | 2011-12-02 | 2020-05-13 | Armagen, Inc. | Methods and compositions for increasing arylsulfatase a activity in the cns |
| US20140377244A1 (en) | 2011-12-23 | 2014-12-25 | Shire Human Genetic Therapies, Inc. | Stable formulations for cns delivery of arylsulfatase a |
| BR112014015352A8 (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 |
| CA2912678C (en) * | 2013-05-15 | 2023-10-10 | Regents Of The University Of Minnesota | Adeno-associated virus mediated gene transfer to the central nervous system |
| US10538589B2 (en) * | 2015-01-14 | 2020-01-21 | Armagen Inc. | Methods and compositions for increasing N-acetylglucosaminidase (NAGLU) activity in the CNS using a fusion antibody comprising an anti-human insulin receptor antibody and NAGLU |
| US10967073B2 (en) | 2015-05-07 | 2021-04-06 | The Mclean Hospital Corporation | Glucocerebrosidase gene therapy for Parkinson's disease |
| CN108430494A (zh) * | 2015-12-30 | 2018-08-21 | 株式会社绿十字 | 用于治疗亨特综合征的方法和组合物 |
| EP3419651B1 (en) | 2016-02-24 | 2023-10-25 | BioMarin Pharmaceutical Inc. | Improved naglu fusion protein formulation |
| IL305449B1 (en) | 2016-04-15 | 2025-09-01 | Univ Pennsylvania | Gene therapy for the treatment of mucocutaneous type ii diabetes |
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