AR120055A1 - Métodos para tratar la enfermedad de fabry en pacientes que tienen insuficiencia renal - Google Patents
Métodos para tratar la enfermedad de fabry en pacientes que tienen insuficiencia renalInfo
- Publication number
- AR120055A1 AR120055A1 ARP200101648A ARP200101648A AR120055A1 AR 120055 A1 AR120055 A1 AR 120055A1 AR P200101648 A ARP200101648 A AR P200101648A AR P200101648 A ARP200101648 A AR P200101648A AR 120055 A1 AR120055 A1 AR 120055A1
- Authority
- AR
- Argentina
- Prior art keywords
- disease
- patients
- methods
- kidney failure
- patient
- Prior art date
Links
- 208000024720 Fabry Disease Diseases 0.000 title abstract 2
- 208000001647 Renal Insufficiency Diseases 0.000 title abstract 2
- 201000006370 kidney failure Diseases 0.000 title abstract 2
- LXBIFEVIBLOUGU-DPYQTVNSSA-N migalastat Chemical compound OC[C@H]1NC[C@H](O)[C@@H](O)[C@H]1O LXBIFEVIBLOUGU-DPYQTVNSSA-N 0.000 abstract 2
- 229950007469 migalastat Drugs 0.000 abstract 2
- GRGNVOCPFLXGDQ-TWHXEDJUSA-N (2r,3r,4s,5r,6r)-2-[(2r,3r,4r,5r,6s)-6-[(2r,3s,4r,5r,6r)-6-[(e,2s,3r)-2-amino-3-hydroxyoctadec-4-enoxy]-4,5-dihydroxy-2-(hydroxymethyl)oxan-3-yl]oxy-4,5-dihydroxy-2-(hydroxymethyl)oxan-3-yl]oxy-6-(hydroxymethyl)oxane-3,4,5-triol Chemical compound O[C@@H]1[C@@H](O)[C@H](OC[C@H](N)[C@H](O)/C=C/CCCCCCCCCCCCC)O[C@H](CO)[C@H]1O[C@H]1[C@H](O)[C@@H](O)[C@@H](O[C@@H]2[C@@H]([C@@H](O)[C@@H](O)[C@@H](CO)O2)O)[C@@H](CO)O1 GRGNVOCPFLXGDQ-TWHXEDJUSA-N 0.000 abstract 1
- 239000012458 free base Substances 0.000 abstract 1
- 150000003839 salts Chemical class 0.000 abstract 1
Classifications
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K31/00—Medicinal preparations containing organic active ingredients
- A61K31/33—Heterocyclic compounds
- A61K31/395—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
- A61K31/435—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with one nitrogen as the only ring hetero atom
- A61K31/44—Non condensed pyridines; Hydrogenated derivatives thereof
- A61K31/445—Non condensed piperidines, e.g. piperocaine
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P13/00—Drugs for disorders of the urinary system
- A61P13/12—Drugs for disorders of the urinary system of the kidneys
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P3/00—Drugs for disorders of the metabolism
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12Y—ENZYMES
- C12Y302/00—Hydrolases acting on glycosyl compounds, i.e. glycosylases (3.2)
- C12Y302/01—Glycosidases, i.e. enzymes hydrolysing O- and S-glycosyl compounds (3.2.1)
- C12Y302/01022—Alpha-galactosidase (3.2.1.22)
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K9/00—Medicinal preparations characterised by special physical form
- A61K9/0012—Galenical forms characterised by the site of application
- A61K9/0053—Mouth and digestive tract, i.e. intraoral and peroral administration
Landscapes
- Health & Medical Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- General Health & Medical Sciences (AREA)
- Organic Chemistry (AREA)
- Medicinal Chemistry (AREA)
- Pharmacology & Pharmacy (AREA)
- Animal Behavior & Ethology (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Engineering & Computer Science (AREA)
- Bioinformatics & Cheminformatics (AREA)
- General Chemical & Material Sciences (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Epidemiology (AREA)
- Urology & Nephrology (AREA)
- Genetics & Genomics (AREA)
- Biochemistry (AREA)
- General Engineering & Computer Science (AREA)
- Wood Science & Technology (AREA)
- Zoology (AREA)
- Hematology (AREA)
- Obesity (AREA)
- Diabetes (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Investigating Or Analysing Biological Materials (AREA)
Abstract
Se proporcionan métodos para al tratamiento de la enfermedad de Fabry en un paciente que tiene insuficiencia renal. Determinados métodos comprenden administrar al paciente de aproximadamente 100 mg a aproximadamente 300 mg de equivalente de base libre de migalastat o sal del mismo a una frecuencia mayor de una vez cada dos días, tal como una vez cada cuatro o siete días. Determinados métodos comprenden medir la liso-Gb3 y/o el migalastat en una o más muestras de plasma del paciente.
Applications Claiming Priority (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201962859904P | 2019-06-11 | 2019-06-11 |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| AR120055A1 true AR120055A1 (es) | 2022-02-02 |
Family
ID=71950725
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| ARP200101648A AR120055A1 (es) | 2019-06-11 | 2020-06-11 | Métodos para tratar la enfermedad de fabry en pacientes que tienen insuficiencia renal |
Country Status (16)
| Country | Link |
|---|---|
| US (1) | US20220313670A1 (es) |
| EP (1) | EP3982962A1 (es) |
| JP (2) | JP7677910B2 (es) |
| KR (1) | KR20220019796A (es) |
| CN (1) | CN114423427A (es) |
| AR (1) | AR120055A1 (es) |
| AU (1) | AU2020291002A1 (es) |
| BR (1) | BR112021024886A2 (es) |
| CA (1) | CA3141226A1 (es) |
| CL (1) | CL2021003280A1 (es) |
| EA (1) | EA202290024A1 (es) |
| IL (1) | IL288677A (es) |
| MX (1) | MX2021015352A (es) |
| PH (1) | PH12021553102A1 (es) |
| TW (1) | TW202112372A (es) |
| WO (1) | WO2020252129A1 (es) |
Families Citing this family (6)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| JP2009537149A (ja) | 2006-05-16 | 2009-10-29 | アミークス セラピューティックス インコーポレイテッド | ファブリー病に対する治療の選択を診断し、評価するためのアッセイ |
| PL3470077T3 (pl) | 2008-02-12 | 2021-04-06 | Amicus Therapeutics, Inc. | Sposób przewidywania odpowiedzi na leczenie chorób chaperonami farmakologicznymi |
| CA3224546A1 (en) | 2017-05-30 | 2018-12-06 | Jeff Castelli | Use of migalastat in the treatment of fabry patients having renal impairment |
| EP3749307B1 (en) | 2018-02-06 | 2025-12-10 | Amicus Therapeutics, Inc. | Use of migalastat for treating fabry disease in pregnant patients |
| JP2021024864A (ja) | 2019-08-07 | 2021-02-22 | アミカス セラピューティックス インコーポレイテッド | Ｇｌａ遺伝子に突然変異を有する患者のファブリー病を治療する方法 |
| US11623916B2 (en) | 2020-12-16 | 2023-04-11 | Amicus Therapeutics, Inc. | Highly purified batches of pharmaceutical grade migalastat and methods of producing the same |
Family Cites Families (10)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US6274597B1 (en) | 1998-06-01 | 2001-08-14 | Mount Sinai School Of Medicine Of New York University | Method of enhancing lysosomal α-Galactosidase A |
| US20100113517A1 (en) | 2007-03-30 | 2010-05-06 | Amicus Therapeutics, Inc. | Method for the treatment of fabry disease using pharmacological chaperones |
| AU2008245578A1 (en) * | 2007-04-26 | 2008-11-06 | Amicus Therapeutics, Inc. | Dosing regimens for the treatment of lysosomal storage diseases using pharmacological chaperones |
| PL3470077T3 (pl) | 2008-02-12 | 2021-04-06 | Amicus Therapeutics, Inc. | Sposób przewidywania odpowiedzi na leczenie chorób chaperonami farmakologicznymi |
| SG193379A1 (en) * | 2011-03-11 | 2013-10-30 | Amicus Therapeutics Inc | Dosing regimens for the treatment of fabry disease |
| EP3487502A1 (en) * | 2016-07-19 | 2019-05-29 | Amicus Therapeutics, Inc. | Treatment of fabry disease in ert-naïve and ert-experienced patients |
| BR112019013920A2 (pt) * | 2017-01-05 | 2020-02-04 | Protalix Ltd | método para tratar doença de fabry, e, forma unitária de dosagem. |
| HUE065615T2 (hu) * | 2017-05-30 | 2024-06-28 | Amicus Therapeutics Inc | Migalasztát, vesekárosodással szövõdött Fabry-kórban szenvedõ betegeg kezelésére |
| AR111971A1 (es) * | 2017-05-30 | 2019-09-04 | Amicus Therapeutics Inc | Métodos para tratar pacientes con enfermedad de fabry que tienen insuficiencia renal |
| CA3224546A1 (en) * | 2017-05-30 | 2018-12-06 | Jeff Castelli | Use of migalastat in the treatment of fabry patients having renal impairment |
-
2020
- 2020-06-11 BR BR112021024886A patent/BR112021024886A2/pt unknown
- 2020-06-11 TW TW109119640A patent/TW202112372A/zh unknown
- 2020-06-11 CA CA3141226A patent/CA3141226A1/en active Pending
- 2020-06-11 PH PH1/2021/553102A patent/PH12021553102A1/en unknown
- 2020-06-11 WO PCT/US2020/037174 patent/WO2020252129A1/en not_active Ceased
- 2020-06-11 JP JP2021573404A patent/JP7677910B2/ja active Active
- 2020-06-11 AU AU2020291002A patent/AU2020291002A1/en active Pending
- 2020-06-11 MX MX2021015352A patent/MX2021015352A/es unknown
- 2020-06-11 AR ARP200101648A patent/AR120055A1/es unknown
- 2020-06-11 EP EP20751716.0A patent/EP3982962A1/en active Pending
- 2020-06-11 CN CN202080042938.3A patent/CN114423427A/zh active Pending
- 2020-06-11 EA EA202290024A patent/EA202290024A1/ru unknown
- 2020-06-11 US US17/618,277 patent/US20220313670A1/en active Pending
- 2020-06-11 KR KR1020227000878A patent/KR20220019796A/ko active Pending
-
2021
- 2021-12-05 IL IL288677A patent/IL288677A/en unknown
- 2021-12-09 CL CL2021003280A patent/CL2021003280A1/es unknown
-
2025
- 2025-05-01 JP JP2025076158A patent/JP2025131572A/ja active Pending
Also Published As
| Publication number | Publication date |
|---|---|
| CA3141226A1 (en) | 2020-12-17 |
| MX2021015352A (es) | 2022-04-06 |
| BR112021024886A2 (pt) | 2022-01-25 |
| JP2025131572A (ja) | 2025-09-09 |
| WO2020252129A1 (en) | 2020-12-17 |
| EP3982962A1 (en) | 2022-04-20 |
| US20220313670A1 (en) | 2022-10-06 |
| JP2022536687A (ja) | 2022-08-18 |
| CL2021003280A1 (es) | 2022-10-07 |
| AU2020291002A1 (en) | 2022-01-06 |
| EA202290024A1 (ru) | 2022-03-14 |
| JP7677910B2 (ja) | 2025-05-15 |
| IL288677A (en) | 2022-02-01 |
| PH12021553102A1 (en) | 2023-10-09 |
| KR20220019796A (ko) | 2022-02-17 |
| TW202112372A (zh) | 2021-04-01 |
| CN114423427A (zh) | 2022-04-29 |
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| AR120055A1 (es) | Métodos para tratar la enfermedad de fabry en pacientes que tienen insuficiencia renal | |
| CL2019003496A1 (es) | Métodos de tratamiento de pacientes con enfermedad de fabry que tienen insuficiencia renal. | |
| MX2023012450A (es) | Esketamina para el tratamiento de la depresion. | |
| AR109103A1 (es) | Tratamiento de la enfermedad de fabry en pacientes no tratados y tratados previamente con ert | |
| AR114392A1 (es) | Tratamiento de pacientes con enfermedad de fabry clásica | |
| MX2017000628A (es) | Metodos para tratar pacientes de alto riesgo cardiovascular con hipercolesterolemia. | |
| CL2024000466A1 (es) | Métodos de tratamiento de la insuficiencia cardíaca mediante la administración de omecamtiv mecarbil | |
| UY35313A (es) | Antiadhesion de leucocitos para mitigar los potenciales eventos adversos causados por los dominios de unión específicos de cd3 | |
| NI201100147A (es) | Métodos para tratar infartos agudos de miocardio y trastornos relacionados. | |
| BR112018002046A2 (pt) | métodos de tratamento da síndrome de lennox-gastaut usando fenfluramina | |
| MX2020006284A (es) | Tetratiomolibdato de bis-colina para tratar la enfermedad de wilson. | |
| AR112482A1 (es) | Métodos para aumentar y/o estabilizar la función cardiaca en pacientes con enfermedad de fabry | |
| MX2018013873A (es) | Irinotecan nanoliposomico para usarse en el tratamiento del cancer de pulmon de celulas pequeñas. | |
| EA202091112A1 (ru) | Сокристаллы, фармацевтические композиции на их основе и способы лечения, предусматривающие их применение | |
| CO2022005113A2 (es) | Métodos de tratamiento para modificar la hemodinámica | |
| EA202090242A1 (ru) | Лечение болезни фабри у фзт-наивных и фзт-леченных пациентов | |
| WO2019010301A8 (en) | Treatment of heart disease by inhibition of the action of muscle a-kinase anchoring protein (makap) | |
| MX2018002816A (es) | Metodos de tratamiento de mieloma multiple y leucemia de celulas plasmaticas mediante terapia de celulas t. | |
| CR20220127A (es) | Administración de dosis para tratamiento con anticuerpos antagonistas anti-tigit y anti-pd-l1 | |
| AR111971A1 (es) | Métodos para tratar pacientes con enfermedad de fabry que tienen insuficiencia renal | |
| EP4327869A3 (en) | Methods of treating fabry patients having renal impairment | |
| MX2022003628A (es) | Regímenes de dosificación para el tratamiento de pacientes con carcinoma de células escamosas avanzado localmente. | |
| EA201992869A1 (ru) | Способы лечения пациентов с болезнью фабри, у которых имеется почечная недостаточность | |
| UA113183C2 (xx) | Спосіб лікування адикції | |
| MX2016011002A (es) | Tratamiento de angioedema hereditario con inhibidor c1. |