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

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

Evaluation of serum MMP-9 as predictive biomarker for antisense therapy in Duchenne

评估血清 MMP-9 作为杜氏肌营养不良症反义疗法的预测生物标志物

A Lourbakos, N Yau, P de Bruijn, M Hiller, K Kozaczynska, R Jean-Baptiste, M Reza, R Wolterbeek, Z Koeks, B Ayoglu, D de Klerk, G Campion, I Zaharieva, V D Nadarajah, P Nilsson, C Al-Khalili Szigyarto, F Muntoni, H Lochmüller, J J Verschuuren, N Goemans, M Tulinius, E H Niks, S de Kimpe, A Aartsma-R

Long-Term Efficacy, Safety, and Pharmacokinetics of Drisapersen in Duchenne Muscular Dystrophy: Results from an Open-Label Extension Study

Drisapersen治疗杜氏肌营养不良症的长期疗效、安全性和药代动力学:一项开放标签扩展研究的结果

Goemans, Nathalie M; Tulinius, Már; van den Hauwe, Marleen; Kroksmark, Anna-Karin; Buyse, Gunnar; Wilson, Rosamund J; van Deutekom, Judith C; de Kimpe, Sjef J; Lourbakos, Afrodite; Campion, Giles