JP2020514305A5 - - Google Patents

Download PDF

Info

Publication number
JP2020514305A5
JP2020514305A5 JP2019538173A JP2019538173A JP2020514305A5 JP 2020514305 A5 JP2020514305 A5 JP 2020514305A5 JP 2019538173 A JP2019538173 A JP 2019538173A JP 2019538173 A JP2019538173 A JP 2019538173A JP 2020514305 A5 JP2020514305 A5 JP 2020514305A5
Authority
JP
Japan
Prior art keywords
pharmaceutical composition
composition according
subject
effective amount
disease
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
Application number
JP2019538173A
Other languages
English (en)
Japanese (ja)
Other versions
JP7576293B2 (ja
JP2020514305A (ja
Filing date
Publication date
Application filed filed Critical
Priority claimed from PCT/US2018/013509 external-priority patent/WO2018132667A1/en
Publication of JP2020514305A publication Critical patent/JP2020514305A/ja
Publication of JP2020514305A5 publication Critical patent/JP2020514305A5/ja
Priority to JP2022181240A priority Critical patent/JP2023011938A/ja
Priority to JP2024159770A priority patent/JP2024169509A/ja
Application granted granted Critical
Publication of JP7576293B2 publication Critical patent/JP7576293B2/ja
Active legal-status Critical Current
Anticipated expiration legal-status Critical

Links

JP2019538173A 2017-01-13 2018-01-12 ファーバー病を治療するための組成物及び方法 Active JP7576293B2 (ja)

Priority Applications (2)

Application Number Priority Date Filing Date Title
JP2022181240A JP2023011938A (ja) 2017-01-13 2022-11-11 ファーバー病を治療するための組成物及び方法
JP2024159770A JP2024169509A (ja) 2017-01-13 2024-09-17 ファーバー病を治療するための組成物及び方法

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201762446166P 2017-01-13 2017-01-13
US62/446,166 2017-01-13
PCT/US2018/013509 WO2018132667A1 (en) 2017-01-13 2018-01-12 Compositions and methods for treating farber disease

Related Child Applications (1)

Application Number Title Priority Date Filing Date
JP2022181240A Division JP2023011938A (ja) 2017-01-13 2022-11-11 ファーバー病を治療するための組成物及び方法

Publications (3)

Publication Number Publication Date
JP2020514305A JP2020514305A (ja) 2020-05-21
JP2020514305A5 true JP2020514305A5 (https=) 2021-02-12
JP7576293B2 JP7576293B2 (ja) 2024-10-31

Family

ID=62839661

Family Applications (3)

Application Number Title Priority Date Filing Date
JP2019538173A Active JP7576293B2 (ja) 2017-01-13 2018-01-12 ファーバー病を治療するための組成物及び方法
JP2022181240A Pending JP2023011938A (ja) 2017-01-13 2022-11-11 ファーバー病を治療するための組成物及び方法
JP2024159770A Pending JP2024169509A (ja) 2017-01-13 2024-09-17 ファーバー病を治療するための組成物及び方法

Family Applications After (2)

Application Number Title Priority Date Filing Date
JP2022181240A Pending JP2023011938A (ja) 2017-01-13 2022-11-11 ファーバー病を治療するための組成物及び方法
JP2024159770A Pending JP2024169509A (ja) 2017-01-13 2024-09-17 ファーバー病を治療するための組成物及び方法

Country Status (8)

Country Link
US (4) US20190343936A1 (https=)
EP (2) EP3568154B1 (https=)
JP (3) JP7576293B2 (https=)
AU (2) AU2018207564A1 (https=)
CA (1) CA3049771A1 (https=)
JO (1) JOP20190164B1 (https=)
MX (1) MX2019008038A (https=)
WO (1) WO2018132667A1 (https=)

Families Citing this family (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
MX2019008038A (es) * 2017-01-13 2020-02-05 Icahn School Med Mount Sinai Composiciones y metodos para tratar la enfermedad de farber.
EP3648776A4 (en) * 2017-07-06 2021-04-07 The Medical College of Wisconsin, Inc. NEW IN-VITRO AND IN-VIVO ENRICHMENT STRATEGY AGAINST LYMPHOCYTE FROM VECTOR-TRANSDUCED HSC FOR THE TREATMENT OF DISEASES
KR20250016468A (ko) 2018-02-02 2025-02-03 엔지반트 테라퓨틱스 게엠베하 파버병을 치료하는 방법
EP3784695B1 (en) 2018-04-27 2023-08-09 The Medical College of Wisconsin, Inc. Use of lentivector-transduced t-rapa cells for amelioration of lysosomal storage disorders
WO2020152532A1 (en) 2019-01-23 2020-07-30 Enzyvant Therapeutics Gmbh Method of ameliorating a pro-inflammatory immunophenotype in farber disease subjects by repeated administration of a recombinant human acid ceramidase
WO2021055801A1 (en) 2019-09-18 2021-03-25 The Medical College Of Wisconsin, Inc. Improved alpha-galactosidase protein for enzyme replacement therapy (ert) and methods of use

Family Cites Families (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
PL2854910T3 (pl) 2012-06-01 2020-07-27 Icahn School Of Medicine At Mount Sinai Poziomy ceramidu w leczeniu i zapobieganiu infekcjom
CA2905449C (en) 2013-03-14 2024-09-10 Icahn School Of Medicine At Mount Sinai THERAPEUTIC COMPOSITIONS OF ACID CERAMIDASE AND THEIR PROCESSES OF MANUFACTURE AND USE
MX2019008038A (es) * 2017-01-13 2020-02-05 Icahn School Med Mount Sinai Composiciones y metodos para tratar la enfermedad de farber.
WO2019060837A1 (en) * 2017-09-25 2019-03-28 Enzyvant Farber Gmbh PROCESS FOR PRODUCING RECOMBINANT HUMAN ACID CERAMIDASE
KR20250016468A (ko) * 2018-02-02 2025-02-03 엔지반트 테라퓨틱스 게엠베하 파버병을 치료하는 방법

Similar Documents

Publication Publication Date Title
JP2020514305A5 (https=)
RU2733464C2 (ru) Препараты tpp1 и способы лечения заболевания cln2
JP2016516016A5 (https=)
TW201521718A (zh) 低頻率格拉替雷(glatiramer)醋酸鹽之治療
FI4017871T3 (fi) Alfa-sarkoglykaaniin liittyvä adeno-assosioidun virusvektorin antaminen ja lihasdystrofian hoito
MX383971B (es) Composición farmacéutica para la prevención y tratamiento de enfermedades del ojo que contiene, como ingrediente activo, una proteína de fusión en la cual el péptido que penetra el tejido y la preparación del factor de crecimiento endotelial antivascular se fusionan.
EP2164500B1 (en) The use of zeburaline for the treatment of autoimmune diseases or immune rejection of transplants
CN103908657A (zh) 胰升糖素样肽-1类似物在制备眼科疾病药物中的用途
US20220280547A1 (en) Compositions and methods for treating long covid
US20130096048A1 (en) Treatment of sepsis and septic shock using ghrelin and growth hormone
WO2011097148A3 (en) Use of oral heparin preparations to treat urinary tract diseases and conditions
JP7074300B2 (ja) ガバペンチノイドからの干渉が減少し、hgf異型体を発現するdnaコンストラクトを用いる神経病症の治療
Wedemeyer et al. 96 weeks of pegylated-Interferon-alpha-2a plus tenofovir or placebo for the treatment of hepatitis delta: the HIDIT-2 study: 31
WO2023122355A2 (en) Methods of treating or preventing infusion-related reactions
JP7684788B2 (ja) 代謝疾患を治療する組成物と方法
AU2013335678A1 (en) Therapeutic agent for amyotrophic lateral sclerosis
US20210236594A1 (en) Methods for improving frailty and aging
Anghel et al. Safety of vascular endothelial and hepatocyte growth factor gene therapy in patients with critical limb ischemia
RU2463045C1 (ru) Способ лечения цереброваскулярных заболеваний и астенического синдрома
CN114588164A (zh) 瑞马唑仑在预防围术期低体温和寒战中的应用
US20230277650A1 (en) Use of a birnavirus for the treatment of a disease caused by varicella zoster virus (vzv)
CN108014337B (zh) 一种用于治疗眼自身免疫性葡萄膜炎的药物组合物
Birettoni et al. Treatment of chronic atrial fibrillation in the horse with flecainide: personal observation.
CN120114599A (zh) Gpr55抑制剂在制备用于增强肿瘤免疫效果的产品中的应用
CN117165587A (zh) 一种miR-99a-5p模拟物及其在制备预防或治疗肝纤维化药物中的应用