日期:
2020 年 — 2026 年
2020
2021
2022
2023
2024
2025
2026
影响因子:

Results of a phase II open-label, multiple-dose study of vamorolone (VBP15-006) in 7- to < 18-year-old boys with duchenne muscular dystrophy

一项针对7至<18岁杜氏肌营养不良症男孩的Vamorolone(VBP15-006)II期开放标签、多剂量研究的结果

Lochmüller, Hanns; Gonorazky, Hernan; Nigro, Elisa; Mah, Jean K; Alemán, Alberto; Yaworski, Amanda; Oskoui, Maryam; Sbrocchi, Anne Marie; Selby, Kathryn; de Vera, Ana; McAdam, Laura; Gresko, Ekaterina; Linden, Aki; Dutreix, Catherine; Hoffman, Eric P

Adrenal Suppression in Duchenne Muscular Dystrophy: Management Strategies Incorporating Novel Steroid Vamorolone

杜氏肌营养不良症的肾上腺抑制:包含新型类固醇Vamorolone的治疗策略

Sbrocchi, Anne Marie; Kinnett, Kathi; Lautatzis, Maria-Elena; McMillan, Hugh J; Selby, Kathryn A; Veerapandiyan, Aravind; Weber, David R; Apkon, Susan; Bharucha-Goebel, Diana X; Bharill, Sonum; Bansal, Shipra; Clemens, Paula R; Fiscaletti, Melissa; Halloun, Rana; Lam, Carol; Merchant, Nadia; McAdam, Laura; Nasomyont, Nat; Nicolau, Stefan; Ochoa Molina, Maria F; Phung, Kim; Rutter, Meilan M; Scavina, Mena; Surampudi, Prasanth N; Tamaroff, Jaclyn; Tian, Cuixia; Ward, Leanne M; Wood, Claire L; Wong, Sze Choong; Ahmet, Alex

Survey of Adrenal Insufficiency Management for Duchenne muscular dystrophy in Italy

意大利杜氏肌营养不良症肾上腺功能不全管理调查

Tornese, Gianluca; Wong, Sze Choong; Sbrocchi, Anne Marie; De Angelis, Fernanda; Zito, Ilaria; Weber, David R; Aversa, Tommaso

Comparison of Transthoracic Echocardiography (TTE) and Transesophageal Echocardiography (TEE) Guidance for Transcatheter Aortic Valve Replacement (TAVR) Performed Under Conscious Sedation: A Retrospective Study

经胸超声心动图(TTE)与经食道超声心动图(TEE)引导下经导管主动脉瓣置换术(TAVR)在清醒镇静下应用的比较:一项回顾性研究

Khan, Ramlah; Ramadan, Emad; Taha, Mohamad; Sbrocchi, Alexander; Banerjee, Subhash; Stoler, Robert C; Ramamoorthy, Saravanan

Consensus-Based Expert Recommendations for Diagnosis and Clinical Management of Vanishing White Matter

基于共识的专家建议:白质消失的诊断和临床管理

van Voorst, Romy J; Schoenmakers, Daphne H; Bonkowsky, Joshua L; Vanderver, Adeline; Krägeloh-Mann, Ingeborg; Bernard, Geneviève; Bertini, Enrico; Fatemi, Ali; Sgobbi, Paulo V; Wolf, Nicole I; Groeschel, Samuel; Tonduti, Davide; Sevin, Caroline; Orthmann-Murphy, Jennifer L; Schöls, Ludger; Salsano, Ettore; Brais, Bernard; Jaffe, Nicole; Ter Horst, Kasper W; Hannema, Sabine E; Hayes, Katherine G; Meyburg, Jochen; van Heerde, Marc; Sbrocchi, Anne Marie; van Spaendonk, Rosalina; Thiffault, Isabelle; Hofsteenge, Geesje H; Sudmeier-Broek, Carolina; Timmer, Corrie; Skwirut, Donna; Buck, Allyson; Hollberg, Bret; Chapleau, Ron; Dekker, Hanka; Campbell, Susan G; Abbink, Truus E M; Leferink, Prisca S; van der Knaap, Marjo S

Exome Sequencing Identifies a Novel Splicing Variant in COL9A3 Resulting in Multiple Epiphyseal Dysplasia: A Case Report

外显子组测序发现COL9A3基因中一种新的剪接变异导致多发性骨骺发育不良:病例报告

Alnuaimi, Bashayer; Miranda, Valancy; Sbrocchi, Anne Marie; Campeau, Philippe M; Campillo, Sarah; Lahiry, Piya

Efficacy and Safety of Vamorolone Over 48 Weeks in Boys With Duchenne Muscular Dystrophy: A Randomized Controlled Trial

Vamorolone治疗杜氏肌营养不良症男孩48周的疗效和安全性:一项随机对照试验

Dang, Utkarsh J; Damsker, Jesse M; Guglieri, Michela; Clemens, Paula R; Perlman, Seth J; Smith, Edward C; Horrocks, Iain; Finkel, Richard S; Mah, Jean K; Deconinck, Nicolas; Goemans, Nathalie M; Haberlová, Jana; Straub, Volker; Mengle-Gaw, Laurel; Schwartz, Benjamin D; Harper, Amy; Shieh, Perry B; De Waele, Liesbeth; Castro, Diana; Yang, Michele L; Ryan, Monique M; McDonald, Craig M; Tulinius, Mar; Webster, Richard I; Mcmillan, Hugh J; Kuntz, Nancy; Rao, Vamshi K; Baranello, Giovanni; Spinty, Stefan; Childs, Anne-Marie; Sbrocchi, Annie M; Selby, Kathryn A; Monduy, Migvis; Nevo, Yoram; Vilchez, Juan J; Nascimento-Osorio, Andres; Niks, Erik H; De Groot, Imelda J M; Katsalouli, Marina; Van Den Anker, John N; Ward, Leanne M; Leinonen, Mika; D'Alessandro, Andrea L; Hoffman, Eric P

Vertebral Body Reshaping after Fractures: An Important Index of Recovery in Glucocorticoid-Treated Children

骨折后椎体重塑:糖皮质激素治疗儿童康复的重要指标

Ma, Jinhui; Siminoski, Kerry; Jaremko, Jacob L; Koujok, Khaldoun; Matzinger, Mary Ann; Shenouda, Nazih; Wilson, Nagwa; Cheng, Megan; Alos, Nathalie; Atkinson, Stephanie; Cummings, Elizabeth A; Ho, Josephine; Rodd, Celia; Sbrocchi, Anne Marie; Stein, Robert; Barr, Ronald; Cairney, Elizabeth; Dix, David B; Fernandez, Conrad V; Grant, Ronald; Halton, Jacqueline; Israels, Sara; Laverdière, Caroline; Lewis, Victor A; Cabral, David A; Huber, Adam; Houghton, Kristin; Jurencak, Roman; Lang, Bianca; Larché, Maggie; LeBlanc, Claire M A; Miettunen, Paivi; Roth, Johannes; Scuccimarri, Rosie; Bell, Lorraine; Blydt-Hansen, Tom; Filler, Guido; Feber, Janusz; Phan, Véronique; Smit, Kevin; Rauch, Frank; Ward, Leanne M

Improved memory CD8 T cell response to delayed vaccine boost is associated with a distinct molecular signature

延迟接种加强疫苗后记忆性CD8 T细胞反应的增强与独特的分子特征相关。

Natalini, Ambra; Simonetti, Sonia; Favaretto, Gabriele; Lucantonio, Lorenzo; Peruzzi, Giovanna; Muñoz-Ruiz, Miguel; Kelly, Gavin; Contino, Alessandra M; Sbrocchi, Roberta; Battella, Simone; Capone, Stefania; Folgori, Antonella; Nicosia, Alfredo; Santoni, Angela; Hayday, Adrian C; Di Rosa, Francesca

Corrigendum: Improved memory CD8 T cell response to delayed vaccine boost is associated with a distinct molecular signature

更正:延迟接种加强疫苗后记忆性 CD8 T 细胞反应的增强与独特的分子特征相关。

Natalini, Ambra; Simonetti, Sonia; Favaretto, Gabriele; Lucantonio, Lorenzo; Peruzzi, Giovanna; Muñoz-Ruiz, Miguel; Kelly, Gavin; Contino, Alessandra M; Sbrocchi, Roberta; Battella, Simone; Capone, Stefania; Folgori, Antonella; Nicosia, Alfredo; Santoni, Angela; Hayday, Adrian C; Di Rosa, Francesca