JP2023159405A5 - - Google Patents
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- Publication number
- JP2023159405A5 JP2023159405A5 JP2023138821A JP2023138821A JP2023159405A5 JP 2023159405 A5 JP2023159405 A5 JP 2023159405A5 JP 2023138821 A JP2023138821 A JP 2023138821A JP 2023138821 A JP2023138821 A JP 2023138821A JP 2023159405 A5 JP2023159405 A5 JP 2023159405A5
- Authority
- JP
- Japan
- Prior art keywords
- composition
- biomarker
- gmfm
- sulfatide
- results
- Prior art date
- Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
- Pending
Links
- 102100022146 Arylsulfatase A Human genes 0.000 claims 6
- 108010036867 Cerebroside-Sulfatase Proteins 0.000 claims 6
- 230000007659 motor function Effects 0.000 claims 5
- 239000000090 biomarker Substances 0.000 claims 4
- 210000001175 cerebrospinal fluid Anatomy 0.000 claims 3
- 230000007423 decrease Effects 0.000 claims 3
- 206010051290 Central nervous system lesion Diseases 0.000 claims 2
- 201000011442 Metachromatic leukodystrophy Diseases 0.000 claims 2
- OTCCIMWXFLJLIA-UHFFFAOYSA-N N-acetyl-DL-aspartic acid Natural products CC(=O)NC(C(O)=O)CC(O)=O OTCCIMWXFLJLIA-UHFFFAOYSA-N 0.000 claims 2
- OTCCIMWXFLJLIA-BYPYZUCNSA-N N-acetyl-L-aspartic acid Chemical group CC(=O)N[C@H](C(O)=O)CC(O)=O OTCCIMWXFLJLIA-BYPYZUCNSA-N 0.000 claims 2
- 238000002595 magnetic resonance imaging Methods 0.000 claims 2
- 239000002207 metabolite Substances 0.000 claims 2
- 230000006641 stabilisation Effects 0.000 claims 2
- 238000011105 stabilization Methods 0.000 claims 2
- BXSULSOCJNTUJS-YTBMLWRQSA-N 1-(3-O-sulfo-beta-D-galactosyl)sphingosine Chemical compound CCCCCCCCCCCCC\C=C\[C@@H](O)[C@@H](N)CO[C@@H]1O[C@H](CO)[C@H](O)[C@H](OS(O)(=O)=O)[C@H]1O BXSULSOCJNTUJS-YTBMLWRQSA-N 0.000 claims 1
- 102000004190 Enzymes Human genes 0.000 claims 1
- 108090000790 Enzymes Proteins 0.000 claims 1
- 230000003044 adaptive effect Effects 0.000 claims 1
- 125000003275 alpha amino acid group Chemical group 0.000 claims 1
- 150000001413 amino acids Chemical class 0.000 claims 1
- 230000001149 cognitive effect Effects 0.000 claims 1
- 230000006870 function Effects 0.000 claims 1
- 238000000185 intracerebroventricular administration Methods 0.000 claims 1
- 238000007913 intrathecal administration Methods 0.000 claims 1
- 238000000034 method Methods 0.000 claims 1
- 238000004611 spectroscopical analysis Methods 0.000 claims 1
- 208000011580 syndromic disease Diseases 0.000 claims 1
- 230000003442 weekly effect Effects 0.000 claims 1
Priority Applications (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2024082556A JP2024102370A (ja) | 2016-02-17 | 2024-05-21 | アリールスルファターゼａのｃｎｓ送達のための方法および組成物 |
Applications Claiming Priority (6)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201662296563P | 2016-02-17 | 2016-02-17 | |
| US62/296,563 | 2016-02-17 | ||
| US201762453864P | 2017-02-02 | 2017-02-02 | |
| US62/453,864 | 2017-02-02 | ||
| JP2018543324A JP2019509270A (ja) | 2016-02-17 | 2017-02-17 | アリールスルファターゼａのｃｎｓ送達のための方法および組成物 |
| JP2021124414A JP2021169528A (ja) | 2016-02-17 | 2021-07-29 | アリールスルファターゼａのｃｎｓ送達のための方法および組成物 |
Related Parent Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2021124414A Division JP2021169528A (ja) | 2016-02-17 | 2021-07-29 | アリールスルファターゼａのｃｎｓ送達のための方法および組成物 |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2024082556A Division JP2024102370A (ja) | 2016-02-17 | 2024-05-21 | アリールスルファターゼａのｃｎｓ送達のための方法および組成物 |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2023159405A JP2023159405A (ja) | 2023-10-31 |
| JP2023159405A5 true JP2023159405A5 (https=) | 2024-05-29 |
Family
ID=58192404
Family Applications (4)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2018543324A Pending JP2019509270A (ja) | 2016-02-17 | 2017-02-17 | アリールスルファターゼａのｃｎｓ送達のための方法および組成物 |
| JP2021124414A Withdrawn JP2021169528A (ja) | 2016-02-17 | 2021-07-29 | アリールスルファターゼａのｃｎｓ送達のための方法および組成物 |
| JP2023138821A Pending JP2023159405A (ja) | 2016-02-17 | 2023-08-29 | アリールスルファターゼａのｃｎｓ送達のための方法および組成物 |
| JP2024082556A Pending JP2024102370A (ja) | 2016-02-17 | 2024-05-21 | アリールスルファターゼａのｃｎｓ送達のための方法および組成物 |
Family Applications Before (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2018543324A Pending JP2019509270A (ja) | 2016-02-17 | 2017-02-17 | アリールスルファターゼａのｃｎｓ送達のための方法および組成物 |
| JP2021124414A Withdrawn JP2021169528A (ja) | 2016-02-17 | 2021-07-29 | アリールスルファターゼａのｃｎｓ送達のための方法および組成物 |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2024082556A Pending JP2024102370A (ja) | 2016-02-17 | 2024-05-21 | アリールスルファターゼａのｃｎｓ送達のための方法および組成物 |
Country Status (10)
| Country | Link |
|---|---|
| US (3) | US11020461B2 (https=) |
| EP (1) | EP3416678A1 (https=) |
| JP (4) | JP2019509270A (https=) |
| CN (2) | CN108883162A (https=) |
| AU (1) | AU2017220100B2 (https=) |
| BR (1) | BR112018016874A2 (https=) |
| CA (1) | CA3014909A1 (https=) |
| MA (1) | MA44237A (https=) |
| MX (2) | MX2018009937A (https=) |
| WO (1) | WO2017143233A1 (https=) |
Families Citing this family (2)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2019126376A1 (en) * | 2017-12-19 | 2019-06-27 | Shire Human Genetic Therapies, Inc. | Purified arylsulfatase a and compositons thereof |
| BR112021021908A2 (pt) * | 2019-05-03 | 2022-02-01 | Univ Pennsylvania | Composições úteis no tratamento da leucodistrofia metacromática |
Family Cites Families (6)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US6217552B1 (en) | 1999-03-01 | 2001-04-17 | Coaxia, Inc. | Medical device for selective intrathecal spinal cooling in aortic surgery and spinal trauma |
| US6537785B1 (en) | 1999-09-14 | 2003-03-25 | Genzyme Glycobiology Research Institute, Inc. | Methods of treating lysosomal storage diseases |
| NZ605874A (en) * | 2010-06-25 | 2015-02-27 | Shire Human Genetic Therapies | Methods and compositions for cns delivery of heparan n-sulfatase |
| SMT201600385T1 (it) * | 2010-06-25 | 2017-03-08 | Shire Human Genetic Therapies | Trasporto di agenti terapeutici all’snc |
| NZ605873A (en) * | 2010-06-25 | 2015-02-27 | Shire Human Genetic Therapies | Methods and compositions for cns delivery of arylsulfatase a |
| CN103179980B (zh) * | 2010-06-25 | 2016-09-28 | 夏尔人类遗传性治疗公司 | 艾杜糖醛酸-2-硫酸酯酶的cns递送的方法和组合物 |
-
2017
- 2017-02-17 EP EP17708395.3A patent/EP3416678A1/en not_active Withdrawn
- 2017-02-17 AU AU2017220100A patent/AU2017220100B2/en active Active
- 2017-02-17 WO PCT/US2017/018440 patent/WO2017143233A1/en not_active Ceased
- 2017-02-17 MX MX2018009937A patent/MX2018009937A/es unknown
- 2017-02-17 JP JP2018543324A patent/JP2019509270A/ja active Pending
- 2017-02-17 US US15/999,472 patent/US11020461B2/en active Active
- 2017-02-17 CA CA3014909A patent/CA3014909A1/en active Pending
- 2017-02-17 BR BR112018016874A patent/BR112018016874A2/pt unknown
- 2017-02-17 MA MA044237A patent/MA44237A/fr unknown
- 2017-02-17 CN CN201780022756.8A patent/CN108883162A/zh active Pending
- 2017-02-17 CN CN202311189440.1A patent/CN117224659A/zh active Pending
-
2018
- 2018-08-16 MX MX2024004034A patent/MX2024004034A/es unknown
-
2021
- 2021-04-30 US US17/246,119 patent/US20210315981A1/en not_active Abandoned
- 2021-07-29 JP JP2021124414A patent/JP2021169528A/ja not_active Withdrawn
-
2023
- 2023-08-29 JP JP2023138821A patent/JP2023159405A/ja active Pending
-
2024
- 2024-05-21 JP JP2024082556A patent/JP2024102370A/ja active Pending
- 2024-09-10 US US18/829,900 patent/US20250099555A1/en active Pending
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