AU2011279158B2 - Method and formulations for treating sialic acid deficiencies - Google Patents
Method and formulations for treating sialic acid deficiencies Download PDFInfo
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- AU2011279158B2 AU2011279158B2 AU2011279158A AU2011279158A AU2011279158B2 AU 2011279158 B2 AU2011279158 B2 AU 2011279158B2 AU 2011279158 A AU2011279158 A AU 2011279158A AU 2011279158 A AU2011279158 A AU 2011279158A AU 2011279158 B2 AU2011279158 B2 AU 2011279158B2
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- Australia
- Prior art keywords
- sialic acid
- extended release
- release formulation
- pharmaceutically acceptable
- acceptable salt
- 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.)
- Ceased
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| PCT/US2011/043910 WO2012009474A1 (en) | 2010-07-13 | 2011-07-13 | Method and formulations for treating sialic acid deficiencies |
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| AU2011279158A1 AU2011279158A1 (en) | 2013-02-21 |
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| US9241896B2 (en) | 2008-12-19 | 2016-01-26 | Ultragenyx Pharmaceutical Inc. | Methods and formulations for treating sialic acid deficiencies |
| JP5777712B2 (ja) | 2010-07-13 | 2015-09-09 | ウルトラジェニックス ファーマシューティカル インコーポレイテッド | シアル酸欠乏症を治療するための方法および製剤 |
| CA2849114C (en) | 2011-10-24 | 2020-12-15 | Ultragenyx Pharmaceutical Inc. | Sialic acid analogs |
| BR112014017587A8 (pt) * | 2012-01-18 | 2017-07-04 | Ultragenyx Pharmaceutical Inc | métodos e formulações para o tratamento de deficiências de ácido siálico |
| WO2014031837A1 (en) * | 2012-08-22 | 2014-02-27 | The Regents Of The University Of California | Compositions and methods for enhancing sialic acid levels in tissue |
| SI3218005T1 (sl) | 2014-11-12 | 2023-06-30 | Seagen Inc. | Z glikanom delujoče spojine, in postopki uporabe |
| AR106025A1 (es) | 2015-09-14 | 2017-12-06 | Ultragenyx Pharmaceutical Inc | Formas cristalinas de ácido siálico o una sal o solvato de este |
| CA3002097A1 (en) | 2015-11-12 | 2017-05-18 | Siamab Therapeutics, Inc. | Glycan-interacting compounds and methods of use |
| EP3541847A4 (en) | 2016-11-17 | 2020-07-08 | Seattle Genetics, Inc. | COMPOUNDS INTERACTING WITH GLYCANE AND METHODS OF USE |
| EP3589319A4 (en) | 2017-03-03 | 2021-07-14 | Seagen Inc. | COMPOUNDS INTERACTING WITH GLYCAN AND METHODS OF USE |
| US20200327425A1 (en) * | 2017-10-31 | 2020-10-15 | Once Labs Inc. | Automated feedback-driven prognosis system and method |
| EP3486326A1 (en) | 2017-11-21 | 2019-05-22 | Jennewein Biotechnologie GmbH | Method for the purification of n-acetylneuraminic acid from a fermentation broth |
| US20200371110A1 (en) * | 2018-01-12 | 2020-11-26 | The Wistar Institute Of Anatomy And Biology | Methods for identifying and treating hiv persistence |
| CN110548012B (zh) * | 2019-08-27 | 2022-05-13 | 嘉必优生物技术(武汉)股份有限公司 | 一种唾液酸片剂及其制备方法 |
| TW202513040A (zh) * | 2023-09-01 | 2025-04-01 | 瑞士商赫孚孟拉羅股份公司 | 製備半固體組成物之新穎方法及其用途 |
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| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2004000366A1 (en) * | 2002-06-21 | 2003-12-31 | Novo Nordisk Health Care Ag | Pegylated factor vii glycoforms |
| WO2008150477A2 (en) * | 2007-05-31 | 2008-12-11 | Government Of The United States Of America As Represented By The Secretary Of The Department Of Health And Human Services National Institutes Of Health | N-acetyl mannosamine as a therapeutic agent |
| WO2009032605A2 (en) * | 2007-08-27 | 2009-03-12 | Massachusetts Institute Of Technology | Bi-functional polymer-attached inhibitors of influenza virus |
Family Cites Families (18)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| JPS6168418A (ja) | 1984-09-11 | 1986-04-08 | Kanto Ishi Pharma Co Ltd | 去痰薬 |
| CA2215978A1 (en) | 1995-04-04 | 1996-10-10 | Wound Healing Of Oklahoma | Cancer treatment by photodynamic therapy, in combination with an immunoadjuvant |
| US5624677A (en) | 1995-06-13 | 1997-04-29 | Pentech Pharmaceuticals, Inc. | Controlled release of drugs delivered by sublingual or buccal administration |
| ATE479425T1 (de) | 1998-04-10 | 2010-09-15 | Mitsubishi Chem Corp | Sialsäure-derivate enthaltende feste dispersion |
| US7238677B2 (en) * | 2003-03-28 | 2007-07-03 | Kimberly-Clark Worldwide, Inc. | Prevention of urogenital infections |
| EP1697481A2 (en) | 2003-12-09 | 2006-09-06 | Spherics, Inc. | Bioadhesive polymers with catechol functionality |
| AU2005328670B2 (en) | 2005-03-03 | 2011-08-11 | Mannatech, Inc. | Methods and compositions for modified release of nutritional supplements |
| US20100226855A1 (en) | 2006-03-02 | 2010-09-09 | Spherics, Inc. | Rate-Controlled Oral Dosage Formulations |
| US9744137B2 (en) | 2006-08-31 | 2017-08-29 | Supernus Pharmaceuticals, Inc. | Topiramate compositions and methods of enhancing its bioavailability |
| WO2009137827A2 (en) | 2008-05-09 | 2009-11-12 | Tiara Pharmaceuticals, Inc. | Controlled release of n-acetylcysteine (nac) for reduction of systemic and/or vascular inflammation |
| KR101116864B1 (ko) * | 2008-08-01 | 2012-02-29 | 주식회사 베네비오 | 고지혈증, 지방간 또는 비만의 예방 또는 치료용 조성물 |
| US20100160363A1 (en) | 2008-12-19 | 2010-06-24 | Aaipharma Services Corp. | Extended-release pharmaceutical formulations |
| US9241896B2 (en) | 2008-12-19 | 2016-01-26 | Ultragenyx Pharmaceutical Inc. | Methods and formulations for treating sialic acid deficiencies |
| US20100159001A1 (en) | 2008-12-19 | 2010-06-24 | Cardinal John R | Extended-Release Pharmaceutical Formulations |
| JP5626734B2 (ja) | 2009-05-15 | 2014-11-19 | 公益財団法人ヒューマンサイエンス振興財団 | Gneタンパク質の機能低下に起因する疾患の治療用医薬剤、食品組成物 |
| JP5777712B2 (ja) | 2010-07-13 | 2015-09-09 | ウルトラジェニックス ファーマシューティカル インコーポレイテッド | シアル酸欠乏症を治療するための方法および製剤 |
| CA2849114C (en) | 2011-10-24 | 2020-12-15 | Ultragenyx Pharmaceutical Inc. | Sialic acid analogs |
| BR112014017587A8 (pt) | 2012-01-18 | 2017-07-04 | Ultragenyx Pharmaceutical Inc | métodos e formulações para o tratamento de deficiências de ácido siálico |
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2011
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- 2011-07-13 US US13/810,068 patent/US9511015B2/en active Active
- 2011-07-13 CA CA2805222A patent/CA2805222A1/en not_active Abandoned
- 2011-07-13 CN CN201810170704.1A patent/CN108606956A/zh active Pending
- 2011-07-13 CN CN2011800439776A patent/CN103370069A/zh active Pending
- 2011-07-13 AU AU2011279158A patent/AU2011279158B2/en not_active Ceased
- 2011-07-13 WO PCT/US2011/043910 patent/WO2012009474A1/en not_active Ceased
- 2011-07-13 KR KR1020137003511A patent/KR20140003380A/ko not_active Ceased
- 2011-07-13 BR BR112013000776A patent/BR112013000776A2/pt not_active IP Right Cessation
- 2011-07-13 EP EP11807478.0A patent/EP2593109A4/en not_active Withdrawn
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- 2016-12-19 US US15/383,682 patent/US20170157160A1/en not_active Abandoned
Patent Citations (3)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2004000366A1 (en) * | 2002-06-21 | 2003-12-31 | Novo Nordisk Health Care Ag | Pegylated factor vii glycoforms |
| WO2008150477A2 (en) * | 2007-05-31 | 2008-12-11 | Government Of The United States Of America As Represented By The Secretary Of The Department Of Health And Human Services National Institutes Of Health | N-acetyl mannosamine as a therapeutic agent |
| WO2009032605A2 (en) * | 2007-08-27 | 2009-03-12 | Massachusetts Institute Of Technology | Bi-functional polymer-attached inhibitors of influenza virus |
Also Published As
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| US9554987B2 (en) | 2017-01-31 |
| KR20140003380A (ko) | 2014-01-09 |
| US9511015B2 (en) | 2016-12-06 |
| US20130273160A1 (en) | 2013-10-17 |
| AU2011279158A1 (en) | 2013-02-21 |
| US20130225513A1 (en) | 2013-08-29 |
| CN103370069A (zh) | 2013-10-23 |
| JP5777712B2 (ja) | 2015-09-09 |
| IL224203B (en) | 2018-06-28 |
| WO2012009474A1 (en) | 2012-01-19 |
| US20170157160A1 (en) | 2017-06-08 |
| CA2805222A1 (en) | 2012-01-19 |
| US20170136051A1 (en) | 2017-05-18 |
| BR112013000776A2 (pt) | 2016-05-24 |
| CN108606956A (zh) | 2018-10-02 |
| EP2593109A1 (en) | 2013-05-22 |
| JP2013531041A (ja) | 2013-08-01 |
| EP2593109A4 (en) | 2014-01-08 |
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