JP2016526050A5 - - Google Patents
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- Publication number
- JP2016526050A5 JP2016526050A5 JP2016518053A JP2016518053A JP2016526050A5 JP 2016526050 A5 JP2016526050 A5 JP 2016526050A5 JP 2016518053 A JP2016518053 A JP 2016518053A JP 2016518053 A JP2016518053 A JP 2016518053A JP 2016526050 A5 JP2016526050 A5 JP 2016526050A5
- Authority
- JP
- Japan
- Prior art keywords
- level
- lyso
- spm
- therapeutic agent
- biological sample
- 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.)
- Granted
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- OKTWQKXBJUBAKS-WQADZSDSSA-N 2-[[(e,2r,3s)-2-amino-3-hydroxyoctadec-4-enoxy]-hydroxyphosphoryl]oxyethyl-trimethylazanium;chloride Chemical compound [Cl-].CCCCCCCCCCCCC\C=C\[C@H](O)[C@H](N)COP(O)(=O)OCC[N+](C)(C)C OKTWQKXBJUBAKS-WQADZSDSSA-N 0.000 claims 65
- 239000003814 drug Substances 0.000 claims 55
- 229940124597 therapeutic agent Drugs 0.000 claims 55
- 239000012472 biological sample Substances 0.000 claims 43
- 108010061312 Sphingomyelin Phosphodiesterase Proteins 0.000 claims 39
- 102000010126 acid sphingomyelin phosphodiesterase activity proteins Human genes 0.000 claims 37
- 238000000034 method Methods 0.000 claims 27
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 claims 26
- 208000035475 disorder Diseases 0.000 claims 26
- 230000000694 effects Effects 0.000 claims 9
- 208000014060 Niemann-Pick disease Diseases 0.000 claims 4
- 238000012423 maintenance Methods 0.000 claims 4
- 239000000523 sample Substances 0.000 claims 4
- 230000002411 adverse Effects 0.000 claims 3
- 239000008280 blood Substances 0.000 claims 2
- 210000004369 blood Anatomy 0.000 claims 2
- 238000002641 enzyme replacement therapy Methods 0.000 claims 2
- 230000035772 mutation Effects 0.000 claims 2
- 238000012360 testing method Methods 0.000 claims 2
- 230000001225 therapeutic effect Effects 0.000 claims 2
- 238000002560 therapeutic procedure Methods 0.000 claims 2
- 231100000331 toxic Toxicity 0.000 claims 2
- 230000002588 toxic effect Effects 0.000 claims 2
- 101000785978 Homo sapiens Sphingomyelin phosphodiesterase Proteins 0.000 claims 1
- 102000011971 Sphingomyelin Phosphodiesterase Human genes 0.000 claims 1
- 230000002378 acidificating effect Effects 0.000 claims 1
- 230000003247 decreasing effect Effects 0.000 claims 1
- 230000002068 genetic effect Effects 0.000 claims 1
- 102000048866 human SMPD1 Human genes 0.000 claims 1
- 238000012544 monitoring process Methods 0.000 claims 1
- 108090000623 proteins and genes Proteins 0.000 claims 1
- 238000012216 screening Methods 0.000 claims 1
- 210000002966 serum Anatomy 0.000 claims 1
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201361832302P | 2013-06-07 | 2013-06-07 | |
| US61/832,302 | 2013-06-07 | ||
| PCT/US2014/041405 WO2014197859A1 (en) | 2013-06-07 | 2014-06-06 | Marker for acid sphingomyelinase disorders and uses thereof |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2018236856A Division JP6835806B2 (ja) | 2013-06-07 | 2018-12-19 | 酸性スフィンゴミエリナーゼ障害のマーカーおよびその使用 |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2016526050A JP2016526050A (ja) | 2016-09-01 |
| JP2016526050A5 true JP2016526050A5 (cg-RX-API-DMAC7.html) | 2017-07-13 |
| JP6457501B2 JP6457501B2 (ja) | 2019-01-23 |
Family
ID=51062988
Family Applications (4)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2016518053A Active JP6457501B2 (ja) | 2013-06-07 | 2014-06-06 | 酸性スフィンゴミエリナーゼ障害のマーカーおよびその使用 |
| JP2018236856A Active JP6835806B2 (ja) | 2013-06-07 | 2018-12-19 | 酸性スフィンゴミエリナーゼ障害のマーカーおよびその使用 |
| JP2021015390A Active JP7414750B2 (ja) | 2013-06-07 | 2021-02-03 | 酸性スフィンゴミエリナーゼ障害のマーカーおよびその使用 |
| JP2023220385A Pending JP2024038145A (ja) | 2013-06-07 | 2023-12-27 | 酸性スフィンゴミエリナーゼ障害のマーカーおよびその使用 |
Family Applications After (3)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2018236856A Active JP6835806B2 (ja) | 2013-06-07 | 2018-12-19 | 酸性スフィンゴミエリナーゼ障害のマーカーおよびその使用 |
| JP2021015390A Active JP7414750B2 (ja) | 2013-06-07 | 2021-02-03 | 酸性スフィンゴミエリナーゼ障害のマーカーおよびその使用 |
| JP2023220385A Pending JP2024038145A (ja) | 2013-06-07 | 2023-12-27 | 酸性スフィンゴミエリナーゼ障害のマーカーおよびその使用 |
Country Status (29)
Families Citing this family (7)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| PL2470199T3 (pl) * | 2009-08-28 | 2019-11-29 | Icahn School Med Mount Sinai | Enzymatyczna terapia zastępcza ze zwiększaniem dawki do leczenia niedoboru kwaśnej sfingomielinazy |
| EP3004896B1 (en) * | 2013-06-07 | 2019-09-18 | Genzyme Corporation | Marker for acid sphingomyelinase disorders and uses thereof |
| JP6984854B2 (ja) * | 2015-10-08 | 2021-12-22 | 国立大学法人千葉大学 | ニーマン・ピック病ｃ型を予防または治療するための医薬組成物 |
| ES2970423T3 (es) * | 2017-08-24 | 2024-05-28 | Genzyme Corp | Esfingomielinasa ácida humana recombinante para su uso en el tratamiento de afecciones óseas anormales en pacientes con deficiencia de esfingomielinasa ácida |
| EP3692070A1 (en) | 2017-10-02 | 2020-08-12 | Denali Therapeutics Inc. | Fusion proteins comprising enzyme replacement therapy enzymes |
| JP7568648B2 (ja) * | 2019-05-31 | 2024-10-16 | ジェンザイム・コーポレーション | 二次元ｌｃ－ｍｓ／ｍｓシステム |
| EP4562432A2 (en) * | 2022-07-28 | 2025-06-04 | Wylder Nation Foundation | Compositions and methods for diagnosing, treating, and preventing lysosomal storage diseases |
Family Cites Families (16)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US4082781A (en) | 1976-06-04 | 1978-04-04 | The United States Of America As Represented By The Department Of Health, Education And Welfare | Synthesis of 2-alkanoylamino-4-nitrophenyl phosphorylcholine-hydroxide |
| US4039388A (en) | 1976-06-04 | 1977-08-02 | The United States Of America As Represented By The Government | Diagnostic test for Niemann-Pick disease |
| US5773278A (en) | 1991-05-03 | 1998-06-30 | Mount Sinai Medical Center | Acid sphingomyelinase gene |
| WO2002018539A2 (en) | 2000-08-25 | 2002-03-07 | Lysometrix Corporation | Method for assaying the activity of lysosomal enzymes |
| US7001994B2 (en) | 2001-01-18 | 2006-02-21 | Genzyme Corporation | Methods for introducing mannose 6-phosphate and other oligosaccharides onto glycoproteins |
| US7615224B2 (en) | 2004-12-02 | 2009-11-10 | Women's And Children's Hospital | Multiplex-bead complex for determination of lysosomal storage disorders |
| JP2006214731A (ja) | 2005-02-01 | 2006-08-17 | Institute Of Physical & Chemical Research | スフィンゴミエリンの検出方法 |
| CN101356283A (zh) | 2005-12-15 | 2009-01-28 | 纽约州立大学研究基金会 | 用于测量血浆和组织鞘磷脂和磷脂酰胆碱的酶促方法 |
| AR059089A1 (es) * | 2006-01-20 | 2008-03-12 | Genzyme Corp | Administracion intraventricular de una enzima para enfermedades de almacenamiento lisosomal |
| US8026099B2 (en) | 2007-07-26 | 2011-09-27 | Washington University | Lipid profile as a biomarker for early detection of neurological disorders |
| US8497122B2 (en) | 2008-04-11 | 2013-07-30 | Washington University | Biomarkers for Niemann-pick C disease and related disorders |
| PL2470199T3 (pl) * | 2009-08-28 | 2019-11-29 | Icahn School Med Mount Sinai | Enzymatyczna terapia zastępcza ze zwiększaniem dawki do leczenia niedoboru kwaśnej sfingomielinazy |
| JP2013002933A (ja) | 2011-06-16 | 2013-01-07 | Hitachi Plant Technologies Ltd | ３次元測位システム |
| CA2854668A1 (en) * | 2011-11-15 | 2013-05-23 | Centogene Ag | Method for the diagnosis of niemann-pick disease |
| TR201906195T4 (tr) * | 2013-05-14 | 2019-05-21 | Centogene Ag | Niemann pıck hastalığının teşhisi için metot. |
| EP3004896B1 (en) * | 2013-06-07 | 2019-09-18 | Genzyme Corporation | Marker for acid sphingomyelinase disorders and uses thereof |
-
2014
- 2014-06-06 EP EP14735056.5A patent/EP3004896B1/en active Active
- 2014-06-06 CN CN201480043918.2A patent/CN105474017A/zh active Pending
- 2014-06-06 HR HRP20192246TT patent/HRP20192246T1/hr unknown
- 2014-06-06 DK DK14735056.5T patent/DK3004896T3/da active
- 2014-06-06 KR KR1020217007269A patent/KR102292298B1/ko active Active
- 2014-06-06 SI SI201431434T patent/SI3004896T1/sl unknown
- 2014-06-06 UA UAA201600158A patent/UA120591C2/uk unknown
- 2014-06-06 RS RS20191627A patent/RS59677B1/sr unknown
- 2014-06-06 ES ES14735056T patent/ES2762608T3/es active Active
- 2014-06-06 NZ NZ754328A patent/NZ754328A/en unknown
- 2014-06-06 US US14/895,472 patent/US10022428B2/en active Active
- 2014-06-06 KR KR1020167000011A patent/KR102228367B1/ko active Active
- 2014-06-06 AU AU2014274670A patent/AU2014274670B2/en active Active
- 2014-06-06 MY MYPI2015704368A patent/MY185990A/en unknown
- 2014-06-06 PT PT147350565T patent/PT3004896T/pt unknown
- 2014-06-06 EA EA201592265A patent/EA035342B1/ru not_active IP Right Cessation
- 2014-06-06 SG SG10201709925SA patent/SG10201709925SA/en unknown
- 2014-06-06 KR KR1020217025969A patent/KR102434244B1/ko active Active
- 2014-06-06 MX MX2015016844A patent/MX370570B/es active IP Right Grant
- 2014-06-06 LT LTEP14735056.5T patent/LT3004896T/lt unknown
- 2014-06-06 MA MA38638A patent/MA38638B1/fr unknown
- 2014-06-06 SG SG11201509622WA patent/SG11201509622WA/en unknown
- 2014-06-06 HU HUE14735056A patent/HUE047863T2/hu unknown
- 2014-06-06 PL PL14735056T patent/PL3004896T3/pl unknown
- 2014-06-06 CA CA2914751A patent/CA2914751C/en active Active
- 2014-06-06 CN CN202210555370.6A patent/CN115128287A/zh active Pending
- 2014-06-06 WO PCT/US2014/041405 patent/WO2014197859A1/en not_active Ceased
- 2014-06-06 EP EP19191053.8A patent/EP3584580A1/en active Pending
- 2014-06-06 JP JP2016518053A patent/JP6457501B2/ja active Active
- 2014-06-06 IL IL284102A patent/IL284102B2/en unknown
-
2015
- 2015-11-23 ZA ZA2015/08630A patent/ZA201508630B/en unknown
- 2015-12-01 PH PH12015502687A patent/PH12015502687A1/en unknown
- 2015-12-04 CR CR20150638A patent/CR20150638A/es unknown
- 2015-12-04 CL CL2015003563A patent/CL2015003563A1/es unknown
- 2015-12-06 IL IL242964A patent/IL242964B/en unknown
-
2018
- 2018-02-09 AU AU2018200977A patent/AU2018200977B2/en active Active
- 2018-06-08 US US16/003,598 patent/US10888607B2/en active Active
- 2018-12-19 JP JP2018236856A patent/JP6835806B2/ja active Active
-
2020
- 2020-10-22 AU AU2020257114A patent/AU2020257114B2/en active Active
- 2020-12-11 US US17/119,035 patent/US11998592B2/en active Active
-
2021
- 2021-02-03 JP JP2021015390A patent/JP7414750B2/ja active Active
-
2022
- 2022-11-29 IL IL298675A patent/IL298675B2/en unknown
-
2023
- 2023-02-23 AU AU2023201062A patent/AU2023201062B2/en active Active
- 2023-12-27 JP JP2023220385A patent/JP2024038145A/ja active Pending
-
2024
- 2024-04-29 US US18/649,453 patent/US20240398907A1/en active Pending
-
2025
- 2025-08-25 AU AU2025220876A patent/AU2025220876A1/en active Pending
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