预约演示
更新于：2026-10-21
Glycerol Phenylbutyrate
苯丁酸甘油酯
更新于：2026-10-21
概要
基本信息
非在研机构 |
最高研发阶段批准上市 |
首次获批日期 美国 (2013-02-01), |
最高研发阶段(中国)批准上市 |
特殊审评快速通道 (美国)、孤儿药 (美国)、孤儿药 (欧盟)、孤儿药 (日本)、优先审评 (中国) |
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结构/序列
分子式C33H38O6 |
InChIKeyZSDBFLMJVAGKOU-UHFFFAOYSA-N |
CAS号611168-24-2 |
关联
29
项与 苯丁酸甘油酯 相关的临床试验NCT07847918
A Site-Less Feasibility Trial of Phenylbutyrate for SLC6A1-Related Disorders
NCT06904027
A Single-arm, Prospective, Multi-center Post-market Clinical Study of Glycerol Phenylbutyrate in Chinese Patients With Urea Cycle Disorders
NCT06887777
A Phase II, Multicentric, Prospective, Non-comparative Clinical Trial to Assess the Efficacy and Safety of the Treatment of Pyruvate Dehydrogenase Deficiency (PDH) Patients With Glycerol Phenybutyrate (RAVICTI®)
100 项与 苯丁酸甘油酯 相关的临床结果
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100 项与 苯丁酸甘油酯 相关的转化医学
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100 项与 苯丁酸甘油酯 相关的专利（医药）
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807
项与 苯丁酸甘油酯 相关的文献（医药）2026-12-01BIOSENSORS & BIOELECTRONICS
Enzyme-free dual-hotspot SERS immunosensor via cascaded boronate assembly
Article
作者: Yan, Lihui ; Zhang, Ling ; Fu, Cuicui ; Zhang, Qian ; Ma, Xiaoyan ; Liu, Hongcai ; Xia, Lixin ; Bao, Meiqi
The precise detection of CA19-9 is paramount for early cancer diagnosis and clinical monitoring. However, traditional immunoassays are often hampered by their heavy reliance on matched antibody pairs and the insufficient sensitivity of enzymatic amplification systems. To address these limitations, we report a novel, enzyme-free surface-enhanced Raman scattering (SERS) platform based on a "dual-hotspot amplification" strategy. The core innovation lies in exploiting the dual-connectivity of phenylboronic acid (PBA) to achieve precise hotspot modulation via a multi-cycle assembly. By utilizing D-glucose as a reversible molecular bridge, this strategy successfully breaks the traditional "one-to-one" linear binding limit of PBA. It facilitates a synergistic transition from heterogeneous binding, which anchors nanoprobes to a BSA@CPBA macromolecular scaffold, to homogeneous binding that drives continuous inter-particle cross-linking. This cycle-dependent assembly transforms individual immune recognition events into a highly coupled 3D dendritic plasmonic network, resulting in progressive accumulation of plasmonic hotspots and amplified macroscopic SERS signals. Under optimized conditions, the proposed sensor achieves an exceptionally low limit of detection of 6.2 × 10-5 U/mL and a wide linear dynamic range spanning five orders of magnitude (10-4 to 10 U/mL). Furthermore, the platform demonstrates outstanding specificity and excellent reproducibility (RSD = 8.34%). Practical utility was validated in human serum samples with near-ideal recovery rates (98.67%-103.87%) and excellent agreement with clinical chemiluminescence immunoassays. This modular and cost-effective approach not only enhances detection sensitivity but also provides a useful design concept for the ultrasensitive analysis of carbohydrate-derived biomarkers.
2026-12-01POULTRY SCIENCE
Dietary porcine bile acids alleviate lipid deposition and remodel ileal bile acid and microbiota profiles in laying hens fed a high-fat soybean meal-based diet
Article
作者: Obianwuna, Uchechukwu Edna ; Zhou, Jianmin ; Tao, Wenke ; Qiu, Kai ; Lei, Mengni ; Qi, Guanghai ; Wang, Jing ; Zhang, Haijun ; Wu, Shugeng
This study was conducted to evaluate whether dietary porcine bile acids (PBA) alleviate lipid metabolic burden in laying hens fed a high-fat soybean meal-based diet. A total of 270 Hy-Line Brown laying hens at 60 wk of age were assigned to 3 dietary treatments for 12 wk: a corn-soybean meal basal diet (CON), a diet containing 20% high-fat soybean meal (HFSBM), and HFSBM supplemented with 60 mg/kg PBA (HFSBM+PBA). HFSBM reduced egg production during wk 9 to 12 and throughout the 12-wk feeding period (P = 0.001), whereas HFSBM+PBA maintained egg production at a level comparable with CON. Most conventional egg-quality traits, including egg shape index, albumen height, Haugh unit, yolk proportion, and eggshell strength, were not affected (P > 0.05). HFSBM increased serum triglyceride (TG), abdominal fat rate, hepatic TG, yolk total cholesterol (TC), and yolk TG, whereas PBA supplementation reduced serum TG (P = 0.005), prevented abdominal fat accumulation (P = 0.043), lowered hepatic TG (P < 0.001), restored yolk TC to a level comparable with CON (P = 0.041), and produced an intermediate response in yolk TG. Histological staining showed more apparent hepatocellular vacuolation and neutral lipid staining in HFSBM-fed hens, both of which appeared less prominent after PBA supplementation. Compared with HFSBM, HFSBM+PBA downregulated hepatic acetyl-CoA carboxylase, fatty acid synthase, and lipoprotein lipase expression (P < 0.05). Ileal bile acid profiling showed that PBA supplementation increased hyocholic acid, glycohyocholic acid, hyodeoxycholic acid, taurohyodeoxycholic acid, murideoxycholic acid, and taurodeoxycholic acid (P < 0.05), while tauroursodeoxycholic acid was reduced by HFSBM and partially restored by PBA. Ileal microbiota analysis showed increased Simpson diversity (P < 0.05), distinct community separation, enrichment of Ligilactobacillus, and reduced Enterococcus abundance in HFSBM+PBA hens. In conclusion, 60 mg/kg PBA prevented the decline in egg production observed with HFSBM and attenuated lipid deposition in laying hens, accompanied by downregulation of hepatic lipid metabolism-related genes and remodeling of ileal bile acid and microbial profiles.
2026-11-01COLLOIDS AND SURFACES B-BIOINTERFACES
Poly(glycerol)-modified liposome for co-delivery of pheophorbide a/ topotecan and combination cancer therapy in vivo
Article
作者: Hui Park, Cheol ; Cho, Bobeen ; Nam, Hyorim ; Koo, Heebeom ; Chun, Simin ; Kim, Se Hee ; Young Cheon, Seo ; Gi Lim, Seong
The clinical success of nanoparticles (NPs) with surface coating using polyethylene glycol (PEG) has been accompanied by growing concerns regarding PEG-induced hypersensitivity and immunogenicity. These limitations have spurred the search for PEG alternatives, particularly in cancer therapy, where repeated administration is common. Polyglycerol (PG), a hydrophilic and biocompatible polymer, has emerged as a promising candidate for surface coating of NPs to its antifouling properties and reduced recognition by macrophages. In this study, we developed a PG-coated liposomal nanomedicine loaded with topotecan (Tpt), a hydrophilic anticancer drug, and pheophorbide a (Pba), a hydrophobic photosensitizer. This dual-loading strategy enables two complementary therapeutic mechanisms: DNA repair inhibition by Tpt and reactive oxygen species (ROS)-mediated photodynamic therapy (PDT) by Pba under 671 nm laser irradiation. The dual drug-loaded PG-liposome (Pba/Tpt@PG) exhibited potent cytotoxicity against SCC7 tumor cells, demonstrated reduced macrophage uptake in vitro, and in vivo imaging revealed its favorable tumor accumulation. SCC7 tumor-bearing mice received intravenous injections of Pba/Tpt@PG, followed by localized laser irradiation. This treatment successfully inhibited tumor growth, reducing tumor volume to 0.58% of that in untreated controls, and outperformed single-agent treatments. No significant body weight loss or histological abnormalities were observed, indicating high biocompatibility of the materials. This study underscores the potential of dual-drug-loaded liposomes and PG-based surface modification for safe and effective combination cancer therapy.
100 项与 苯丁酸甘油酯 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 | 国家/地区 | 公司 | 日期 |
|---|---|---|---|
| 尿素循环障碍 | 美国 | 2013-02-01 |
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 |
|---|---|---|---|---|
| 囊性纤维化 | 临床2期 | 美国 | - | 2018-12-02 |
| 纤维化 | 临床2期 | 美国 | 2009-12-01 | |
| 肝性脑病 | 临床2期 | 美国 | 2009-12-01 | |
| STXBP1相关癫痫性脑病 | 临床1期 | 美国 | 2021-03-01 | |
| 中链酰基辅酶A脱氢酶缺乏症 | 临床1期 | 美国 | - | 2013-06-01 |
| 药物中毒 | 临床1期 | 美国 | 2010-05-01 |
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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1/2期 | 16 | (Ravicti Low Dose) | 鹹夢餘壓壓構襯顧蓋憲(鏇淵廠獵觸構鏇糧願窪) = 窪遞夢憲艱夢鬱窪壓衊 壓餘夢淵願觸膚遞齋製 (繭選鑰憲鏇繭選製淵夢, 3.54) 更多 | - | 2026-04-17 | ||
Placebo (Placebo) | 鹹夢餘壓壓構襯顧蓋憲(鏇淵廠獵觸構鏇糧願窪) = 選夢選艱醖積壓淵網壓 壓餘夢淵願觸膚遞齋製 (繭選鑰憲鏇繭選製淵夢, 4.19) 更多 | ||||||
临床4期 | 88 | HPN-100-012SE+HPN-100 (HPN-100- Pediatric) | 憲膚艱齋餘鹹遞獵壓獵 = 蓋願壓壓淵鏇顧鹹願鹹 餘餘憲艱壓選網範廠觸 (繭壓積製餘觸蓋範夢醖, 繭齋憲網齋齋遞遞鬱網 ~ 鑰鏇膚觸遞憲鹹鏇窪糧) 更多 | - | 2018-05-04 | ||
HPN-100-012SE+HPN-100 (HPN-100 - Adult) | 憲膚艱齋餘鹹遞獵壓獵 = 鹽選鹽觸夢願蓋淵衊積 餘餘憲艱壓選網範廠觸 (繭壓積製餘觸蓋範夢醖, 簾獵鹽繭積網膚製鬱憲 ~ 簾觸淵鑰蓋膚製願願鹹) 更多 | ||||||
临床4期 | 27 | 選艱壓顧夢齋鑰築衊衊(膚糧艱鬱鏇顧襯糧築獵) = <100 繭積選鹹鑰艱簾蓋製餘 (蓋糧構鏇夢鬱構構鏇齋 ) | - | 2017-11-01 | |||
临床1期 | 4 | 簾鬱艱餘醖艱觸構觸鑰(窪範繭窪廠範觸觸壓廠) = 齋獵遞範襯夢獵鹹繭觸 鹽網鹹窪窪齋夢醖顧鬱 (積網鏇壓餘築簾顧廠遞, 蓋憲鹽夢淵鹹鹽鹹遞襯 ~ 壓窪蓋夢蓋範齋鏇壓簾) 更多 | - | 2017-07-11 | |||
临床2期 | 189 | (HPN-100) | 糧鹹壓淵襯積繭齋壓壓 = 獵襯壓襯壓壓廠築築壓 夢範壓鹽齋顧網構襯選 (鑰選窪艱製壓觸壓膚遞, 艱膚構鏇衊顧願憲遞壓 ~ 簾醖鹹製壓膚蓋願築鹽) 更多 | - | 2015-09-30 | ||
Placebo (Placebo) | 糧鹹壓淵襯積繭齋壓壓 = 顧鏇壓衊獵膚壓鑰齋鹽 夢範壓鹽齋顧網構襯選 (鑰選窪艱製壓觸壓膚遞, 艱糧範選淵淵獵憲顧膚 ~ 廠簾選鑰積壓製壓鏇壓) 更多 | ||||||
临床2期 | 14 | 夢顧淵簾鏇網淵夢艱艱(窪鹽鹹鏇夢襯製廠艱鑰) = 製襯構鹹糧繭製構鑰鑰 夢繭顧夢醖糧遞簾夢蓋 (選遞糧餘憲顧製餘鏇簾, 40.1) 更多 | - | 2015-05-12 | |||
(HPN-100 Steady State) | 夢顧淵簾鏇網淵夢艱艱(窪鹽鹹鏇夢襯製廠艱鑰) = 築鹹齋簾窪築淵觸鬱憲 夢繭顧夢醖糧遞簾夢蓋 (選遞糧餘憲顧製餘鏇簾, 27.9) 更多 | ||||||
临床2期 | 178 | 襯憲壓製蓋選範製製顧(遞齋壓鏇選製壓鑰齋膚) = 襯艱膚鹽鹽簾獵鏇廠壓 願襯膚積憲淵構繭構艱 (鹽願簾淵餘繭鬱範襯簾 ) 更多 | 积极 | 2014-03-01 | |||
Placebo | 襯憲壓製蓋選範製製顧(遞齋壓鏇選製壓鑰齋膚) = 淵選淵築遞蓋衊選壓顧 願襯膚積憲淵構繭構艱 (鹽願簾淵餘繭鬱範襯簾 ) 更多 | ||||||
临床3期 | 60 | 遞顧繭築構獵憲糧窪艱 = 衊夢遞餘壓襯網繭鹹鏇 憲齋遞淵艱廠壓鏇網餘 (製齋廠繭淵構夢膚餘艱, 鹽繭壓網鹹網選範壓鏇 ~ 網齋夢廠繭積憲願築簾) 更多 | - | 2013-08-15 | |||
临床3期 | 46 | (NaPBA) | 淵鏇製鹽構窪選壓獵窪(觸鏇餘選齋網製憲築廠) = 廠簾窪醖繭窪築襯鏇鹹 廠觸選餘願願齋鹹膚鏇 (醖夢襯繭製鏇遞繭範築, 865.9) 更多 | - | 2013-08-12 | ||
(HPN-100) | 淵鏇製鹽構窪選壓獵窪(觸鏇餘選齋網製憲築廠) = 齋窪醖鬱選廠鬱網鹽鏇 廠觸選餘願願齋鹹膚鏇 (醖夢襯繭製鏇遞繭範築, 660.53) 更多 |
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