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

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

Efficacy and Safety of Vamorolone vs Placebo and Prednisone Among Boys With Duchenne Muscular Dystrophy: A Randomized Clinical Trial

Vamorolone 与安慰剂和泼尼松治疗杜氏肌营养不良症男孩的疗效和安全性:一项随机临床试验

Guglieri, Michela; Clemens, Paula R; Perlman, Seth J; Smith, Edward C; Horrocks, Iain; Finkel, Richard S; Mah, Jean K; Deconinck, Nicolas; Goemans, Nathalie; Haberlova, Jana; Straub, Volker; Mengle-Gaw, Laurel J; Schwartz, Benjamin D; Harper, Amy D; Shieh, Perry B; De Waele, Liesbeth; Castro, Diana; Yang, Michelle L; Ryan, Monique M; McDonald, Craig M; Tulinius, Mar; Webster, Richard; McMillan, Hugh J; Kuntz, Nancy L; Rao, Vashmi K; Baranello, Giovanni; Spinty, Stefan; Childs, Anne-Marie; Sbrocchi, Annie M; Selby, Kathryn A; Monduy, Migvis; Nevo, Yoram; Vilchez-Padilla, Juan J; Nascimento-Osorio, Andres; Niks, Erik H; de Groot, Imelda J M; Katsalouli, Marina; James, Meredith K; van den Anker, Johannes; Damsker, Jesse M; Ahmet, Alexandra; Ward, Leanne M; Jaros, Mark; Shale, Phil; Dang, Utkarsh J; Hoffman, Eric P

Efficacy and Safety of Vamorolone in Duchenne Muscular Dystrophy: A 30-Month Nonrandomized Controlled Open-Label Extension Trial

Vamorolone治疗杜氏肌营养不良症的疗效和安全性:一项为期30个月的非随机对照开放标签扩展试验

Mah, Jean K; Clemens, Paula R; Guglieri, Michela; Smith, Edward C; Finkel, Richard S; Tulinius, Mar; Nevo, Yoram; Ryan, Monique M; Webster, Richard; Castro, Diana; Kuntz, Nancy L; McDonald, Craig M; Damsker, Jesse M; Schwartz, Benjamin D; Mengle-Gaw, Laurel J; Jackowski, Stefan; Stimpson, Georgia; Ridout, Deborah A; Ayyar-Gupta, Vandana; Baranello, Giovanni; Manzur, Adnan Y; Muntoni, Francesco; Gordish-Dressman, Heather; Leinonen, Mika; Ward, Leanne M; Hoffman, Eric P; Dang, Utkarsh J

Vamorolone, a dissociative steroidal compound, reduces collagen antibody-induced joint damage and inflammation when administered after disease onset

Vamorolone是一种分离性甾体化合物,在疾病发作后给药可减轻胶原抗体引起的关节损伤和炎症。

Damsker, Jesse M; Cornish, Michaelyn R; Kanneboyina, Priya; Kanneboyina, Ila; Yu, Qing; Lipson, Rachel; Phadke, Aditi; Knoblach, Susan M; Panchapakesan, Karuna; Morales, Melissa; Fiorillo, Alyson A; Partridge, Terence; Nagaraju, Kanneboyina

Vamorolone targets dual nuclear receptors to treat inflammation and dystrophic cardiomyopathy

Vamorolone靶向双重核受体,用于治疗炎症和营养不良性心肌病。

Heier, Christopher R; Yu, Qing; Fiorillo, Alyson A; Tully, Christopher B; Tucker, Asya; Mazala, Davi A; Uaesoontrachoon, Kitipong; Srinivassane, Sadish; Damsker, Jesse M; Hoffman, Eric P; Nagaraju, Kanneboyina; Spurney, Christopher F

Phase IIa trial in Duchenne muscular dystrophy shows vamorolone is a first-in-class dissociative steroidal anti-inflammatory drug

杜氏肌营养不良症的IIa期临床试验表明,vamorolone是一种首创的解离性甾体抗炎药。

Conklin, Laurie S; Damsker, Jesse M; Hoffman, Eric P; Jusko, William J; Mavroudis, Panteleimon D; Schwartz, Benjamin D; Mengle-Gaw, Laurel J; Smith, Edward C; Mah, Jean K; Guglieri, Michela; Nevo, Yoram; Kuntz, Nancy; McDonald, Craig M; Tulinius, Mar; Ryan, Monique M; Webster, Richard; Castro, Diana; Finkel, Richard S; Smith, Andrea L; Morgenroth, Lauren P; Arrieta, Adrienne; Shimony, Maya; Jaros, Mark; Shale, Phil; McCall, John M; Hathout, Yetrib; Nagaraju, Kanneboyina; van den Anker, John; Ward, Leanne M; Ahmet, Alexandra; Cornish, Michaelyn R; Clemens, Paula R

The corticosteroid compounds prednisolone and vamorolone do not alter the nociception phenotype and exacerbate liver injury in sickle cell mice

皮质类固醇化合物泼尼松龙和伐莫罗龙不会改变镰状细胞小鼠的伤害感受表型,反而会加重其肝损伤。

Almeida, Luis E F; Damsker, Jesse M; Albani, Sarah; Afsar, Nina; Kamimura, Sayuri; Pratt, Drew; Kleiner, David E; Quezado, Martha; Gordish-Dressman, Heather; Quezado, Zenaide M N

Muscle miRNAome shows suppression of chronic inflammatory miRNAs with both prednisone and vamorolone

肌肉 miRNA 组显示,泼尼松和伐莫罗龙均可抑制慢性炎症 miRNA。

Fiorillo, Alyson A; Tully, Christopher B; Damsker, Jesse M; Nagaraju, Kanneboyina; Hoffman, Eric P; Heier, Christopher R

Phase 1 trial of vamorolone, a first-in-class steroid, shows improvements in side effects via biomarkers bridged to clinical outcomes

一项针对首创类固醇药物vamorolone的I期临床试验显示,通过与临床结果相联系的生物标志物,副作用有所改善。

Hoffman, Eric P; Riddle, Valerie; Siegler, Maxime A; Dickerson, Daniel; Backonja, Miroslav; Kramer, William G; Nagaraju, Kanneboyina; Gordish-Dressman, Heather; Damsker, Jesse M; McCall, John M

Serum biomarkers of glucocorticoid response and safety in anti-neutrophil cytoplasmic antibody-associated vasculitis and juvenile dermatomyositis

抗中性粒细胞胞浆抗体相关性血管炎和幼年皮肌炎中糖皮质激素反应和安全性的血清生物标志物

Conklin, Laurie S; Merkel, Peter A; Pachman, Lauren M; Parikh, Hemang; Tawalbeh, Shefa; Damsker, Jesse M; Cuthbertson, David D; Morgan, Gabrielle A; Monach, Paul A; Hathout, Yetrib; Nagaraju, Kanneboyina; van den Anker, John; McAlear, Carol A; Hoffman, Eric P