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 From Vamorolone and Prednisone in Duchenne Muscular Dystrophy: Results From the Phase 2b Clinical Trial

Vamorolone 和泼尼松治疗杜氏肌营养不良症引起的肾上腺抑制:2b 期临床试验结果

Ahmet, Alexandra; Tobin, Rebecca; Dang, Utkarsh J; Rooman, Raoul; Guglieri, Michela; Clemens, Paula R; Hoffman, Eric P; Ward, Leanne M

Circulating protein biomarkers identified in two independent clinical trial cohorts of glucocorticoid-naive Duchenne muscular dystrophy patients

在两项独立的临床试验队列中,对未接受糖皮质激素治疗的杜氏肌营养不良症患者进行了循环蛋白生物标志物的鉴定。

Ahmadiharchegani, Fatemeh; Tobin, Rebecca A; Degan, Chiara; Naveed, Ahmed; Guglieri, Michela; Jiménez-Requena, Albert; de Vries, Sharon I; Szigyarto, Cristina Al-Khalili; Spitali, Pietro; Tsonaka, Roula; van der Burgt, Yuri E M; Diaz-Manera, Jordi; Clemens, Paula R; Hoffman, Eric P; Dang, Utkarsh J; Hathout, Yetrib

The Persistence of Duchenne vs Becker Muscular Dystrophies: Vive la Difference?

杜氏肌营养不良症与贝克尔肌营养不良症的持续性:区别万岁?

Hoffman, Eric P

Association of DMD Gene Variant Classes With Motor Outcomes in a Drug Registration Clinical Trial Setting

DMD基因变异类型与药物注册临床试验环境中的运动功能结局的关联

Fang, Yuan; Dang, Utkarsh J; Illei, Katherine I; Clemens, Paula; Hoffman, Eric P

Test-Retest Reliability of Motor Function and Myometry Outcomes From the Vamorolone Trials in Duchenne Muscular Dystrophy

杜氏肌营养不良症患者接受Vamorolone治疗后运动功能和肌电图结果的重测信度

Tobin, Rebecca A; Hoffman, Eric P; Johnson, Linda; James, Meredith K; Clemens, Paula R; Dang, Utkarsh J

The effect of IL-1β inhibitor canakinumab (Ilaris®) on IL-6 production in human skeletal muscle cells.

IL-1β抑制剂卡那单抗(Ilaris®)对人类骨骼肌细胞中IL-6产生的影响

Cordeiro-Santanach Anna, Morales Fiorella, Parquet Maria Del Carmen, Uaesoontrachoon Kitipong, Rowsell Joyce, Warford Jordan, Wu Wilson, Elustondo Pia, Hoffman Eric P, Nagaraju Kanneboyina

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

Diagnostic outcome of pro bono neurogenetic diagnostic service in Sri Lanka: A wealth creation

斯里兰卡公益神经遗传诊断服务的诊断结果:创造财富

Gonawala, Lakmal; Wijekoon, Nalaka; Attanayake, Darshika; Ratnayake, Pyara; Sirisena, Darshana; Gunasekara, Harsha; Dissanayake, Athula; Keshavaraj, Ajantha; Mohan, Chandra; Steinbusch, Harry W M; Hoffman, Eric P; Dalal, Ashwin; de Silva, K Ranil D

Serum metabolomic signatures of patients with rare neurogenetic diseases: an insight into potential biomarkers and treatment targets

罕见神经遗传疾病患者血清代谢组学特征:揭示潜在生物标志物和治疗靶点

Wijekoon, Nalaka; Gonawala, Lakmal; Ratnayake, Pyara; Sirisena, Darshana; Gunasekara, Harsha; Dissanayake, Athula; Amaratunga, Dhammika; Steinbusch, Harry W M; Hathout, Yetrib; Hoffman, Eric P; Dalal, Ashwin; Mohan, Chandra; de Silva, K Ranil D