预约演示
更新于：2025-04-17
Forodesine Hydrochloride
呋咯地辛
更新于：2025-04-17
概要
基本信息
药物类型 小分子化药 |
别名 (1S)-1,4-dideoxy-4-imino-(9-deazahypoxanthin-9-yl)-D-ribitol、1,4-Dideoxy-4-aza-1-(S)-(9-deazahypoxanthin-9-yl)-D-ribitol、FDS + [10] |
靶点 |
作用方式 抑制剂 |
作用机制 PNP抑制剂(嘌呤核苷磷酸化酶抑制剂)、免疫抑制剂 |
在研适应症 |
在研机构 |
最高研发阶段批准上市 |
首次获批日期 日本 (2017-03-31), |
最高研发阶段(中国)- |
特殊审评孤儿药 (欧盟)、孤儿药 (日本) |
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结构/序列
分子式C11H15ClN4O4 |
InChIKeyWEIAMZKHBCLFOG-QPAIBFMUSA-N |
CAS号284490-13-7 |
关联
21
项与 呋咯地辛 相关的临床试验NCT01776411
Phase I/II Clinical Study of Forodesine in Japanese Recurrent/Refractory Peripheral T-cell Lymphoma Patients
JPRN-JapicCTI-121985
Phase 1/2 clinical study of forodesine in Japanese recurrent/refractory peripheral T-cell lymphoma
JPRN-jRCT2080221940
Phase 1/2 clinical study of forodesine in Japanese recurrent/refractory peripheral T-cell lymphoma
100 项与 呋咯地辛 相关的临床结果
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100 项与 呋咯地辛 相关的转化医学
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100 项与 呋咯地辛 相关的专利（医药）
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278
项与 呋咯地辛 相关的文献（医药）2025-03-01Journal of neurointerventional surgery
Braid stability after flow diverter treatment of intracranial aneurysms: a systematic review and meta-analysis
Review
作者: Saatci, Isil ; Hanel, Ricardo A ; Lylyk, Ivan ; Mendes Pereira, Vitor ; Lamin, Saleh ; Gounis, Matthew J ; Vivanco-Suarez, Juan ; Rice, Hal ; Rodriguez-Calienes, Aaron ; Lylyk, Pedro ; Fiehler, Jens ; Baltacioglu, Feyyaz ; Dibas, Mahmoud ; Cekirge, H Saruhan ; Fiorella, David ; Ortega-Gutierrez, Santiago
Background:
The aim of this study was to evaluate the overall rates of braid changes associated with flow diverter (FD) treatment for intracranial aneurysms (IAs). Additionally, we sought to provide an overview of the currently reported definitions related to these complications.
Methods:
A systematic search was conducted from the inception of relevant literature up to April 2023, encompassing six databases. The included studies focused on patients with IAs treated with FDs. We considered four main outcome measures as FD braid changes: (1) fish-mouthing, (2) device braid narrowing, (3) device braid collapsing, and (4) device braid deformation. The data from these studies were pooled using a random-effects model.
Results:
A total of 48 studies involving 3572 patients were included in the analysis. Among them, 14 studies (39%) provided definitions for fish-mouthing. However, none of the included studies offered specific definitions for device braid narrowing, collapsing, or deformation, despite reporting rates for these complications in six, five, and three studies, respectively. The pooled rates for braid changes were as follows: 3% (95% CI 2% to 4%, I2=27%) for fish-mouthing, 7% (95% CI 2% to 20%, I2=85%) for narrowing, 1% (95% CI 0% to 3%, I2=0%) for collapsing, and 1% (95% CI 1% to 4%, I2=0%) for deformation.
Conclusion:
The findings of this study suggest that FD treatment for IAs generally exhibits low rates of fish-mouthing, device braid narrowing, collapsing, and deformation. However, the lack of standardized definitions hinders the ability to compare device outcomes objectively, emphasizing the need for uniform definitions for FD braid changes in future prospective studies on FD.
2024-12-01ANNALS OF NEUROLOGY
Guanylate Kinase 1 Deficiency: A Novel and Potentially Treatable Mitochondrial DNA Depletion/Deletions Disease
Article
作者: Marti, Ramon ; Meneri, Megi ; Pesini, Alba ; Tort, Frederic ; Sciacco, Monica ; Hirano, Michio ; Milisenda, Jose Cesar ; Koganti, Lahari ; Tanji, Kurenai ; Tadesse, Saba ; Wang, Tian ; Matas‐Garcia, Ana ; Ramon, Javier ; Ugarteburu, Olatz ; Saneto, Russell P. ; Mansukhani, Mahesh ; Gu, Yue ; Shintaku, Jonathan ; Comi, Giacomo Pietro ; Hidalgo‐Gutierrez, Agustin ; Horwitz, Marshall S. ; Ribes, Antonia ; Garrabou, Gloria ; Gort, Laura ; Barriocanal‐Casado, Eliana ; Dorschner, Michael O. ; Ronchi, Dario ; Wang, Shuang
Objective:
Mitochondrial DNA (mtDNA) depletion/deletions syndrome (MDDS) comprises a group of diseases caused by primary autosomal defects of mtDNA maintenance. Our objective was to study the etiology of MDDS in 4 patients who lack pathogenic variants in known genetic causes.
Methods:
Whole exome sequencing of the probands was performed to identify pathogenic variants. We validated the mitochondrial defect by analyzing mtDNA, mitochondrial dNTP pools, respiratory chain activities, and GUK1 activity. To confirm pathogenicity of GUK1 deficiency, we expressed 2 GUK1 isoforms in patient cells.
Results:
We identified biallelic GUK1 pathogenic variants in all 4 probands who presented with ptosis, ophthalmoparesis, and myopathic proximal limb weakness, as well as variable hepatopathy and altered T‐lymphocyte profiles. Muscle biopsies from all probands showed mtDNA depletion, deletions, or both, as well as reduced activities of mitochondrial respiratory chain enzymes. GUK1 encodes guanylate kinase, originally identified as a cytosolic enzyme. Long and short isoforms of GUK1 exist. We observed that the long isoform is intramitochondrial and the short is cytosolic. In probands’ fibroblasts, we noted decreased GUK1 activity causing unbalanced mitochondrial dNTP pools and mtDNA depletion in both replicating and quiescent fibroblasts indicating that GUK1 deficiency impairs de novo and salvage nucleotide pathways. Proband fibroblasts treated with deoxyguanosine and/or forodesine, a purine phosphatase inhibitor, ameliorated mtDNA depletion, indicating potential pharmacological therapies.
Interpretation:
Primary GUK1 deficiency is a new and potentially treatable cause of MDDS. The cytosolic isoform of GUK1 may contribute to the T‐lymphocyte abnormality, which has not been observed in other MDDS disorders. ANN NEUROL 2024;96:1209–1224
2024-11-01Journal of neurointerventional surgery
Two year follow-up of distal unruptured intracranial aneurysms treated with a surface modified flow diverter under prasugrel monotherapy
Article
作者: Freitas, Rafael Kiyuze ; Machado, João Paulo ; Abud, Daniel Giansante ; Nakiri, Guilherme Seizem ; Monsignore, Lucas Moretti ; Abud, Thiago Giansante ; Colli, Benedicto Oscar ; de Oliveira, Ricardo Santos ; de Castro-Afonso, Luis Henrique
Background:
A new generation of modified surface flow diverters (FDs) and monotherapy using new antiplatelets may reduce both ischemic and hemorrhagic complications during the treatment of intracranial aneurysms. Previous preliminary safety analysis of distal unruptured intracranial aneurysms treated with the FD p48 MW HPC (phenox-Wallaby, Bochum, Germany) under antiplatelet monotherapy with prasugrel showed promising results. However, the long term outcomes of distal intracranial aneurysms treated with FDs under antiplatelet monotherapy are not known.
Methods:
This was a single center, prospective, pivotal, open single arm study. The primary (safety) endpoint was absence of any new neurological deficits after treatment until the 24 month follow-up. The primary (efficacy) endpoint was the incidence of complete aneurysm occlusion 24 months after treatment. The secondary (efficacy) endpoints were any incidence of aneurysm dome reduction 24 months after treatment.
Results:
21 patients harboring 27 distal aneurysms of the anterior circulation were included. No patient had neurologic deficits in the time from treatment to the 24 month follow-up. Complete aneurysm occlusion occurred in 20 (74%) of 27 aneurysms at the 24 month follow-up. Four aneurysms (14.8%) had dome reduction, and three aneurysms (11.1%) remained unchanged.
Conclusions:
In this pilot trial, treatment of distal unruptured intracranial aneurysms with an FD under monotherapy with prasugrel, followed by monotherapy with aspirin, appeared to be safe and effective. Randomized studies with long term follow-up are needed to confirm these results.
1
项与 呋咯地辛 相关的新闻（医药）2023-02-01
·生物谷
细胞疗法免疫疗法
100 项与 呋咯地辛 相关的药物交易
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研发状态
批准上市
10 条最早获批的记录, 后查看更多信息
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| 适应症 | 国家/地区 | 公司 | 日期 |
|---|---|---|---|
| 外周T细胞淋巴瘤 | 日本 | 2017-03-31 |
未上市
10 条进展最快的记录, 后查看更多信息
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| 适应症 | 最高研发状态 | 国家/地区 | 公司 | 日期 |
|---|---|---|---|---|
| 前体B细胞急性淋巴细胞白血病 | 临床2期 | 奥地利 | 2009-03-01 | |
| 前体B细胞急性淋巴细胞白血病 | 临床2期 | 捷克 | 2009-03-01 | |
| 前体B细胞急性淋巴细胞白血病 | 临床2期 | 法国 | 2009-03-01 | |
| 前体B细胞急性淋巴细胞白血病 | 临床2期 | 德国 | 2009-03-01 | |
| 前体B细胞急性淋巴细胞白血病 | 临床2期 | 意大利 | 2009-03-01 | |
| 前体B细胞急性淋巴细胞白血病 | 临床2期 | 英国 | 2009-03-01 | |
| 难治性 T 急性淋巴细胞白血病 | 临床2期 | 奥地利 | 2009-03-01 | |
| 难治性 T 急性淋巴细胞白血病 | 临床2期 | 捷克 | 2009-03-01 | |
| 难治性 T 急性淋巴细胞白血病 | 临床2期 | 法国 | 2009-03-01 | |
| 难治性 T 急性淋巴细胞白血病 | 临床2期 | 德国 | 2009-03-01 |
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临床结果
临床结果
适应症
分期
评价
查看全部结果
临床2期 | 外周T细胞淋巴瘤 二线 | 48 | 製淵網製網選選糧壓艱(蓋鑰遞膚選鏇憲鏇顧衊) = 醖鏇鑰窪簾鹽衊築構範 鹽範遞齋鹹淵積鏇選鏇 (顧鑰遞窪襯襯襯鹹積夢, 14 ~ 38) 更多 | 积极 | 2019-01-01 | ||
临床1/2期 | 44 | 遞觸積遞鹹襯構蓋範餘(築餘鑰簾鹹窪觸醖繭憲) = 繭網網壓蓋餘醖夢選艱 顧簾顧餘範膚襯襯鹹範 (壓願壓簾艱鬱艱製鬱窪, 12.0 ~ 35.3) 更多 | 积极 | 2016-05-20 | |||
临床2期 | 144 | 願簾獵齋淵鏇網築鏇窪(製鹽鹹醖醖範糧網觸繭) = 製壓遞築選鏇遞窪觸鑰 構壓淵顧齋觸積選獵構 (構製繭憲鏇顧廠衊網憲 ) 更多 | - | 2014-09-01 | |||
临床2期 | 8 | Oral forodesine 200 mg/d | 襯繭憲齋夢醖壓觸鹹鬱(製襯鑰遞襯膚遞鹹鑰壓) = 襯築鏇淵憲膚遞構艱鑰 夢壓鏇蓋觸鑰窪鹽襯獵 (襯鏇製積鑰夢餘艱繭觸 ) 更多 | - | 2010-08-12 | ||
N/A | 64 | 網遞蓋願觸鏇衊積襯鹹(觸糧糧壓築製淵鑰簾鏇) = 餘淵憲願積鹹繭遞廠衊 憲築繭遞遞觸積觸蓋網 (鹹構壓構廠壓觸網繭鹹 ) 更多 | - | 2009-05-20 |
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