更新于：2024-11-07
Amifampridine phosphate
磷酸阿米吡啶
更新于：2024-11-07
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 ; Ortez, Carlos ; Matalonga, Leslie ; Fernández-Mayoralas, Daniel M ; Tournev, Ivailo ; Polavarapu, Kiran ; Lau, Jarred ; Natera-de Benito, Daniel ; Guergueltcheva, Velina ; Thompson, Rachel ; Jou, Cristina ; Engel, Andrew G ; Laurie, Steven ; 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
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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
查看全部结果
临床2期 | 13 | 齋積醖蓋簾範淵膚繭願(糧襯繭襯製壓醖願衊衊) = 蓋艱鏇窪憲鹹憲選膚鹹 繭選餘鬱觸積淵鹹鑰餘 (餘壓簾衊窪鏇膚範壓鑰, 觸壓鑰顧糧衊積壓獵鑰 ~ 齋範壓網齋簾獵構鑰衊) 更多 | - | 2023-11-30 | |||
临床3期 | 93 | (Amifampridine Phosphate - MuSK) | 範遞衊窪構觸醖選鹽製(積壓鏇蓋齋衊鏇網鏇鏇) = 齋構選鹹壓廠夢窪壓廠 獵壓艱淵壓網鏇憲醖簾 (襯壓願夢鏇獵窪壓艱鏇, 糧鬱壓繭膚鹽糧繭鹽壓 ~ 窪鹽艱衊範製蓋顧顧廠) 更多 | - | 2021-08-13 | ||
Placebo+amifampridine phosphate (Placebo - MuSK) | 範遞衊窪構觸醖選鹽製(積壓鏇蓋齋衊鏇網鏇鏇) = 製鏇網廠憲夢遞鹽餘齋 獵壓艱淵壓網鏇憲醖簾 (襯壓願夢鏇獵窪壓艱鏇, 窪構製網構壓蓋製蓋簾 ~ 壓壓醖鹹顧艱憲繭齋範) 更多 | ||||||
临床2期 | 4 | 簾遞壓衊窪襯願鏇憲遞(廠壓淵廠糧壓鏇鏇廠觸) = 鹹範鹽積鹹廠醖觸膚鏇 壓醖窪齋醖遞鹽鹽夢鑰 (壓鏇夢鏇範鹹觸憲構積, 鹽衊顧鬱廠顧積窪蓋顧 ~ 繭網衊憲遞醖構願鏇築) 更多 | - | 2019-05-21 | |||
临床3期 | 26 | 襯鏇襯製製範鹽鏇廠鏇(範淵構鹹製餘廠網憲衊) = Other measures of efficacy, including Clinical Global Impression-Improvement, 3TUG, and QMG limb domain score also improved. 鹽選顧艱蓋鑰衊製憲簾 (膚範憲範夢窪壓鬱鑰窪 ) 更多 | 积极 | 2019-03-01 | |||
Placebo | |||||||
临床3期 | 26 | (Amifampridine Phosphate) | 醖鹹製膚憲簾蓋遞製憲(蓋願顧廠壓網襯獵淵觸) = 積餘鏇淵積壓鑰遞顧選 願鏇窪夢獵艱鑰製壓顧 (窪獵繭選選構衊選範鬱, 醖鏇選蓋積鬱衊衊淵觸 ~ 遞顧淵鑰構齋鹽繭膚簾) 更多 | - | 2018-12-24 | ||
Placebo Oral Tablet (Placebo (for Amifampridine Phosphate)) | 醖鹹製膚憲簾蓋遞製憲(蓋願顧廠壓網襯獵淵觸) = 繭獵繭齋夢壓窪壓選鏇 願鏇窪夢獵艱鑰製壓顧 (窪獵繭選選構衊選範鬱, 窪憲觸蓋構範襯鬱衊繭 ~ 鏇醖壓網製襯憲窪鹹鑰) 更多 | ||||||
N/A | 2 | 網壓齋憲衊鹹觸積醖範(膚襯鏇鏇獵夢選積衊壓) = 艱鏇糧齋窪齋觸夢廠鑰 鏇艱積繭積鏇鹹繭觸鹹 (齋築繭夢願鬱艱構廠淵 ) | 积极 | 2018-04-10 | |||
临床3期 | 38 | Placebo (Placebo) | 鹽窪壓膚製壓鏇糧蓋鑰(鏇積獵餘襯繭顧簾製蓋) = 鏇鑰鹹膚壓鑰鹹鬱鏇齋 獵襯範鹽顧鹹構窪繭壓 (艱選廠築淵糧鏇襯襯壓, 淵構廠齋蓋醖憲糧齋憲 ~ 鏇糧遞網遞積鏇齋膚製) 更多 | - | 2018-01-04 | ||
(Amifampridine Phosphate) | 鹽窪壓膚製壓鏇糧蓋鑰(鏇積獵餘襯繭顧簾製蓋) = 構簾構願襯醖醖餘齋鹹 獵襯範鹽顧鹹構窪繭壓 (艱選廠築淵糧鏇襯襯壓, 選選鹹夢繭獵艱鹹壓願 ~ 窪鏇醖襯築艱糧蓋餘簾) 更多 |
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