ES2541933T3 - Método para predecir la respuesta al tratamiento farmacológico con chaperona de enfermedades - Google Patents
Método para predecir la respuesta al tratamiento farmacológico con chaperona de enfermedades Download PDFInfo
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- ES2541933T3 ES2541933T3 ES09710178.6T ES09710178T ES2541933T3 ES 2541933 T3 ES2541933 T3 ES 2541933T3 ES 09710178 T ES09710178 T ES 09710178T ES 2541933 T3 ES2541933 T3 ES 2541933T3
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| EP2946785B1 (en) | 2008-02-12 | 2018-10-24 | Amicus Therapeutics, Inc. | Method to predict response to pharmacological chaperone treatment of diseases |
| JP2011517556A (ja) * | 2008-03-12 | 2011-06-16 | アミカス セラピューティックス インコーポレイテッド | ポンペ病のための治療選択肢を診断し評価するための検定 |
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| CN114540327B (zh) | 2014-09-30 | 2026-03-24 | 阿米库斯治疗学公司 | 具有增强的碳水化合物的高强度酸性α-葡糖苷酶 |
| WO2016149508A1 (en) * | 2015-03-19 | 2016-09-22 | Shire Human Genetic Therapies, Inc. | Mrna therapy for pompe disease |
| GB201508025D0 (en) | 2015-05-11 | 2015-06-24 | Ucl Business Plc | Fabry disease gene therapy |
| KR20250007693A (ko) | 2015-12-30 | 2025-01-14 | 아미쿠스 세라퓨틱스, 인코포레이티드 | 폼페병 치료용의 강화된 산 알파-글루코시다제 |
| HRP20231465T1 (hr) | 2015-12-30 | 2024-03-01 | Amicus Therapeutics, Inc. | Poboljšana kisela alfa-glukozidaza za liječenje pompeove bolesti |
| KR20190087673A (ko) * | 2016-03-22 | 2019-07-24 | 아미쿠스 세라퓨틱스, 인코포레이티드 | Ｇｌａ 유전자에 ｇ9331ａ 돌연변이를 갖는 환자에서 파브리 질병의 치료 방법 |
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| CA3019354A1 (en) * | 2016-03-30 | 2017-10-05 | Amicus Therapeutics, Inc. | Method for selection of high m6p recombinant proteins |
| AU2017239641A1 (en) | 2016-03-30 | 2018-10-18 | Amicus Therapeutics, Inc. | Formulations comprising recombinant acid alpha-glucosidase |
| TW202408513A (zh) | 2016-07-19 | 2024-03-01 | 美商阿米庫斯醫療股份有限公司 | 治療ert初治的和已經歷ert的患者中之法布瑞氏症 |
| JP6546135B2 (ja) | 2016-08-24 | 2019-07-17 | 株式会社ノリタケカンパニーリミテド | 積層造形焼成体、積層造形焼成体の製造方法および積層造形焼成体製造用キット |
| KR102712926B1 (ko) * | 2016-10-20 | 2024-10-07 | 상가모 테라퓨틱스, 인코포레이티드 | 파브리병의 치료를 위한 방법 및 조성물 |
| EA039750B1 (ru) * | 2017-03-30 | 2022-03-10 | Амикус Терапьютикс, Инк. | Способ отбора рекомбинантных белков с высоким содержанием m6p |
| SMT202300187T1 (it) | 2017-05-15 | 2023-07-20 | Amicus Therapeutics Inc | Alfa-glucosidasi acida umana ricombinante |
| RS65230B1 (sr) | 2017-05-30 | 2024-03-29 | Amicus Therapeutics Inc | Migalastat za lečenje pacijenata koji boluju od fabrijeve bolesti sa oštećenjem bubrega |
| CA3224546C (en) | 2017-05-30 | 2026-02-24 | Amicus Therapeutics, Inc. | Use of migalastat in the treatment of fabry patients |
| SG11202000423XA (en) | 2017-07-19 | 2020-02-27 | Amicus Therapeutics Inc | Treatment of fabry disease in ert-naïve and ert-experienced patients |
| US20200222377A1 (en) | 2017-08-28 | 2020-07-16 | Amicus Therapeutics, Inc. | Methods Of Enhancing And/Or Stabilizing Cardiac Function In Patients With Fabry Disease |
| PT3749308T (pt) | 2018-02-06 | 2024-01-25 | Amicus Therapeutics Inc | Tratamento de pacientes com doença de fabry clássica com migalastato |
| KR20200128676A (ko) | 2018-02-06 | 2020-11-16 | 아미쿠스 세라퓨틱스, 인코포레이티드 | 임신 환자의 파브리병 치료를 위한 미갈라스타트의 용도 |
| US20220184185A1 (en) | 2018-07-25 | 2022-06-16 | Modernatx, Inc. | Mrna based enzyme replacement therapy combined with a pharmacological chaperone for the treatment of lysosomal storage disorders |
| US12594268B2 (en) | 2018-08-20 | 2026-04-07 | Amicus Therapeutics, Inc. | Methods of treating Fabry disease in patients having a mutation in the GLA gene |
| NL2021840B1 (en) | 2018-10-19 | 2020-05-13 | Univ Leiden | Pharmacological Chaperones For Enzyme Treatment Therapy |
| WO2020046132A1 (en) | 2018-08-31 | 2020-03-05 | Leiden University | Pharmacological chaperones for enzyme treatment therapy |
| TW202042812A (zh) | 2019-01-22 | 2020-12-01 | 美商阿米庫斯醫療股份有限公司 | 減少法布瑞氏症患者中之腦血管事件之方法 |
| MX2021015352A (es) | 2019-06-11 | 2022-04-06 | Amicus Therapeutics Inc | Métodos para tratar la enfermedad de fabry en pacientes que tienen insuficiencia renal. |
| KR20220044560A (ko) * | 2019-08-07 | 2022-04-08 | 아미쿠스 세라퓨틱스, 인코포레이티드 | Gla 유전자에 돌연변이를 갖는 환자에서 파브리 질병을 치료하는 방법 |
| EP4025242A1 (en) | 2019-09-06 | 2022-07-13 | Amicus Therapeutics, Inc. | Method for capturing and purification of biologics |
| KR20220150902A (ko) | 2020-02-10 | 2022-11-11 | 아미쿠스 세라퓨틱스, 인코포레이티드 | 파브리 질병을 치료하는 방법 |
| AU2021230371A1 (en) * | 2020-03-06 | 2022-09-29 | Amicus Therapeutics, Inc. | Methods of treating Fabry disease in patients having a mutation in the GLA gene |
| US11623916B2 (en) | 2020-12-16 | 2023-04-11 | Amicus Therapeutics, Inc. | Highly purified batches of pharmaceutical grade migalastat and methods of producing the same |
| CA3225511A1 (en) | 2021-07-12 | 2023-01-19 | Franklin Johnson | Methods of treating fabry disease in pediatric patients |
| EP4518885A1 (en) | 2022-05-05 | 2025-03-12 | Amicus Therapeutics, Inc. | Methods for treating pompe disease |
| WO2024119091A1 (en) | 2022-12-02 | 2024-06-06 | Amicus Therapeutics, Inc. | Fexamethods for treating infantile-onset pompe disease in pediatric patients |
| AR131256A1 (es) | 2022-12-02 | 2025-03-05 | Amicus Therapeutics Inc | Métodos para tratar la enfermedad de pompe de inicio tardío en pacientes pediátricos |
| US20240197706A1 (en) | 2022-12-13 | 2024-06-20 | Amicus Therapeutics, Inc. | Methods of improving the pharmacokinetics of migalastat |
| WO2026006625A1 (en) | 2024-06-26 | 2026-01-02 | Amicus Therapeutics, Inc. | Miglustat formulations comprising nitrosamine for use in the treatment of pompe disease |
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