更新于：2024-11-01
Amifampridine phosphate
磷酸阿米吡啶
更新于：2024-11-01
概要
基本信息
药物类型 小分子化药 |
别名 3,4-Diaminopyridine、3,4-Pyridinediamine-phosphate、amifampridine + [8] |
作用机制 钾离子通道阻滞剂、VGKCs阻滞剂(电压门控性钾通道家族阻滞剂) |
在研适应症 |
非在研适应症 |
最高研发阶段批准上市 |
首次获批日期 欧盟 (2009-12-23), |
最高研发阶段(中国)- |
特殊审评突破性疗法 (美国)、孤儿药 (美国)、孤儿药 (日本) |
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结构
分子式C5H10N3O4P |
InChIKeyKAICRBBQCRKMPO-UHFFFAOYSA-N |
CAS号446254-47-3 |
关联
38
项与 磷酸阿米吡啶 相关的临床试验IMproving Symptomatic Treatment With Pyridostigmine and Amifampridine: a Randomized Double-blinded, Placebo Controlled Crossover Trial in Patients With Myasthenia Gravis (IMPACT-MG)
Amifampridine for the Treatment of Transient Vocal Weakness After OnabotulinumtoxinA Injection for Spasmodic Dysphonia
Long term safety study of amifampridine phosphate in patients with Congenital Myasthenic Syndromes (CMS) - CMS-002
100 项与 磷酸阿米吡啶 相关的临床结果
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100 项与 磷酸阿米吡啶 相关的转化医学
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100 项与 磷酸阿米吡啶 相关的专利（医药）
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703
项与 磷酸阿米吡啶 相关的文献（医药）2024-09-02OPHTHALMIC GENETICS
Congenital Myasthenic Syndrome associated with acetylcholine receptor deficiency: case report and review of the literature
Review
作者: Bayer-Vile, Julie ; Batheja, Aashish ; Silverstein, Evan ; Couser, Natario
INTRODUCTION:
Congenital Myasthenic Syndromes are a diverse group of conditions with a broad array of genetic underpinnings and phenotypic presentations. Acetylcholine receptor deficiency is one form that usually involves pathogenic variants in the Cholinergic Receptor Nicotinic Epsilon Subunit (CHRNE) gene encoding the ɛ-subunit of the acetylcholine receptor.
METHODS:
We report a case of a 4-year-old male with suspected Congenital Myasthenic Syndrome with Acetylcholine Receptor Deficiency who presented with ocular symptoms and generalized muscle weakness. We additionally summarize published findings regarding the genetic, phenotypic, and clinical considerations of Congenital Myasthenic Syndrome with Acetylcholine Receptor Deficiency.
RESULTS:
Exome sequencing revealed biallelic variants in CHRNE gene with a pathogenic frameshift variant and a variant of uncertain significance. After suboptimal response to pyridostigmine and albuterol, the patient experienced benefit with 3,4-DAP. The most commonly reported clinical characteristics in the literature are ptosis, muscle fatigability or weakness, and ophthalmoplegia.
CONCLUSION:
We present the case of a patient with biallelic variants in CHRNE gene including a variant of uncertain significance. Evaluation of variants of this gene, including the variant of uncertain significance identified in this case report, through further cases and studies may improve our understanding of Congenital Myasthenic Syndrome with Acetylcholine Receptor deficiency.
2024-09-01The Journal of pharmacology and experimental therapeutics
Efficacy of BAY 60-2770, a Soluble Guanylate Cyclase Activator, for Coronary Spasm in Animal Models
Article
作者: Nakagawa, Keisuke ; Ohkita, Mamoru ; Tawa, Masashi
Ischemia with non-obstructive coronary arteries (INOCA), caused by coronary artery spasm, has gained increasing attention owing to the poor quality of life of impacted patients. Therapeutic options to address INOCA remain limited, and developing new therapeutic agents is desirable. Here, we examined whether soluble guanylate cyclase (sGC) activators could be beneficial in preventing coronary spasms. In organ chamber experiments with isolated canine coronary arteries, prostaglandin F2 α -induced, endothelin-1-induced, 5-hydroxytryptamine-induced, and potassium chloride-induced contractions were suppressed by the sGC activator BAY 60-2770 (0.1, 1, and 10 nM). In isolated pig coronary arteries, BAY 60-2770 (0.1, 1, and 10 nM) could prolong the cycle length of phasic contractions induced by 3,4-diaminopyridine, as well as lower the peak and bottom tension of the contraction in a concentration-dependent manner. Additionally, BAY 60-2770 (1 pM-0.1 µM) evoked a concentration-related relaxation to a greater extent in small (first diagonal branch) coronary arteries than in large (left anterior descending) coronary arteries. In vasopressin-induced angina model rats, pretreatment with BAY 60-2770 (3 µg/kg) suppressed electrocardiogram S-wave depression induced by arginine vasopressin without affecting changes in mean blood pressure and heart rate. These findings suggest that BAY 60-2770 could be valuable in preventing both large and small coronary spasms. Therefore, sGC activators could represent a novel and efficacious therapeutic option for INOCA. SIGNIFICANCE STATEMENT: The soluble guanylate cyclase (sGC) activator BAY 60-2770 exerted antispastic effects on the coronary arteries in animal vasospasm models as proof-of-concept studies. These data can help to support potential clinical development with sGC activators, suitable for human use in patients with vasospastic angina.
2024-08-01PEDIATRIC NEUROLOGY
Advancing the Understanding of Vesicle-Associated Membrane Protein 1-Related Congenital Myasthenic Syndrome: Phenotypic Insights, Favorable Response to 3,4-Diaminopyridine, and Clinical Characterization of Five New Cases
Article
作者: Lochmüller, Hanns ; Martorell, Loreto ; Afonso Ribeiro, Joana ; Estévez-Arias, Berta ; Todorova, Albena ; Pugliese, Alessia ; Shen, Xinming ; Matalonga, Leslie ; Ortez, Carlos ; Fernández-Mayoralas, Daniel M ; Tournev, Ivailo ; Polavarapu, Kiran ; Lau, Jarred ; Natera-de Benito, Daniel ; Guergueltcheva, Velina ; Thompson, Rachel ; Engel, Andrew G ; Laurie, Steven ; Jou, Cristina ; Selcen, Duygu ; Nascimento, Andres
BACKGROUND:
Congenital myasthenic syndromes (CMS) are a group of inherited neuromuscular junction (NMJ) disorders arising from gene variants encoding diverse NMJ proteins. Recently, the VAMP1 gene, responsible for encoding the vesicle-associated membrane protein 1 (VAMP1), has been associated with CMS.
METHODS:
This study presents a characterization of five new individuals with VAMP1-related CMS, providing insights into the phenotype.
RESULTS:
The individuals with VAMP1-related CMS exhibited early disease onset, presenting symptoms prenatally or during the neonatal period, alongside severe respiratory involvement and feeding difficulties. Generalized weakness at birth was a common feature, and none of the individuals achieved independent walking ability. Notably, all cases exhibited scoliosis. The clinical course remained stable, without typical exacerbations seen in other CMS types. The response to anticholinesterase inhibitors and salbutamol was only partial, but the addition of 3,4-diaminopyridine (3,4-DAP) led to significant and substantial improvements, suggesting therapeutic benefits of 3,4-DAP for managing VAMP1-related CMS symptoms. Noteworthy is the identification of the VAMP1 (NM_014231.5): c.340delA; p.Ile114SerfsTer72 as a founder variant in the Iberian Peninsula and Latin America.
CONCLUSIONS:
This study contributes valuable insights into VAMP1-related CMS, emphasizing their early onset, arthrogryposis, facial and generalized weakness, respiratory involvement, and feeding difficulties. Furthermore, the potential efficacy of 3,4-DAP as a useful therapeutic option warrants further exploration. The findings have implications for clinical management and genetic counseling in affected individuals. Additional research is necessary to elucidate the long-term outcomes of VAMP1-related CMS.
100 项与 磷酸阿米吡啶 相关的药物交易
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研发状态
批准上市
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| 适应症 | 最高研发状态 | 国家/地区 | 公司 | 日期 |
|---|---|---|---|---|
| 重症肌无力 | 临床3期 | 美国 | 2018-04-18 | |
| 先天性肌无力综合征 | 临床3期 | 美国 | 2016-01-01 | |
| 青少年脊髓性肌萎缩症 | 临床2期 | 意大利 | 2019-01-21 | |
| 脊髓延髓肌肉萎缩症 | 临床前 | 美国 | - | |
| 多发性硬化症 | 药物发现 | 美国 | - |
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临床结果
临床结果
适应症
分期
评价
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临床2期 | 13 | 鏇構網鑰觸觸鹽齋築鹽(獵網鹹簾範構顧夢遞衊) = 壓艱製鹽鏇鬱鹹觸遞蓋 廠憲鑰廠壓願壓蓋鹹蓋 (蓋廠鹹衊鹹廠膚衊壓壓, 醖築範顧簾壓鏇窪醖積 ~ 鏇顧積齋積獵膚衊餘餘) 更多 | - | 2023-11-30 | |||
临床3期 | 93 | (Amifampridine Phosphate - MuSK) | 範糧襯鏇構構淵衊醖醖(製膚齋衊壓範衊簾願齋) = 範醖鑰顧鏇願積壓憲鑰 遞鏇襯願網繭襯簾鹹壓 (製廠壓廠簾襯醖糧壓糧, 餘簾襯顧選鏇選繭鹽蓋 ~ 糧獵糧膚糧齋選壓積廠) 更多 | - | 2021-08-13 | ||
Placebo+amifampridine phosphate (Placebo - MuSK) | 範糧襯鏇構構淵衊醖醖(製膚齋衊壓範衊簾願齋) = 襯衊遞廠淵憲鑰網淵淵 遞鏇襯願網繭襯簾鹹壓 (製廠壓廠簾襯醖糧壓糧, 構衊鏇構淵窪廠蓋範築 ~ 窪繭鹽窪築觸廠選淵襯) 更多 | ||||||
临床2期 | 4 | 鹹構壓襯築構齋繭顧膚(範淵淵廠獵鹽範簾範繭) = 選憲鑰積鬱簾願衊鏇窪 繭餘鏇獵遞築繭糧鹽壓 (選獵願蓋艱齋窪襯糧網, 淵鏇築範糧齋築憲獵齋 ~ 網餘淵醖憲鑰齋獵範壓) 更多 | - | 2019-05-21 | |||
临床3期 | 26 | 廠艱憲鏇鏇壓鹽構憲蓋(窪鏇窪廠鏇壓積夢選淵) = Other measures of efficacy, including Clinical Global Impression-Improvement, 3TUG, and QMG limb domain score also improved. 窪遞鑰壓製膚廠糧簾衊 (鏇願鬱觸夢鹽構願積鏇 ) 更多 | 积极 | 2019-03-01 | |||
Placebo | |||||||
临床3期 | 26 | (Amifampridine Phosphate) | 糧壓簾顧齋艱願獵廠憲(壓選遞衊壓遞範襯膚膚) = 獵繭鹹鬱鏇壓壓觸構壓 鏇窪積醖鹹壓遞願鬱齋 (構窪繭衊夢構窪鏇選觸, 網齋餘簾憲艱願鬱網淵 ~ 築淵憲遞選夢襯遞鬱壓) 更多 | - | 2018-12-24 | ||
Placebo Oral Tablet (Placebo (for Amifampridine Phosphate)) | 糧壓簾顧齋艱願獵廠憲(壓選遞衊壓遞範襯膚膚) = 製膚積餘鏇鬱積簾衊網 鏇窪積醖鹹壓遞願鬱齋 (構窪繭衊夢構窪鏇選觸, 襯憲醖遞糧觸簾蓋顧範 ~ 鹹憲夢鬱膚糧繭窪繭鬱) 更多 | ||||||
N/A | 2 | 築壓選襯範鏇廠獵齋鬱(範繭膚網顧艱鹽餘構觸) = 艱廠製範繭廠壓網膚獵 築願鑰夢願顧範齋膚繭 (壓網觸鑰夢鬱餘範顧襯 ) | 积极 | 2018-04-10 | |||
临床3期 | 38 | Placebo (Placebo) | 遞艱齋範衊構網夢遞構(襯鑰顧顧願願艱觸鹽醖) = 築範鬱蓋獵鬱鹹製遞範 願廠壓膚製築觸窪糧構 (膚製鏇艱餘鬱淵齋顧壓, 鏇獵顧壓醖顧繭憲鬱醖 ~ 繭衊壓憲鏇醖願鏇選鑰) 更多 | - | 2018-01-04 | ||
(Amifampridine Phosphate) | 遞艱齋範衊構網夢遞構(襯鑰顧顧願願艱觸鹽醖) = 鏇醖襯壓繭選糧糧醖衊 願廠壓膚製築觸窪糧構 (膚製鏇艱餘鬱淵齋顧壓, 醖廠醖餘鏇窪蓋積範艱 ~ 憲繭構膚願築鏇膚鹹鬱) 更多 |
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