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

Rycal S48168 (ARM210) for RYR1-related myopathies: a phase one, open-label, dose-escalation trial

Rycal S48168 (ARM210) 用于治疗 RYR1 相关肌病:一项 I 期、开放标签、剂量递增试验

Todd, Joshua J; Lawal, Tokunbor A; Chrismer, Irene C; Kokkinis, Angela; Grunseich, Christopher; Jain, Minal S; Waite, Melissa R; Biancavilla, Victoria; Pocock, Shavonne; Brooks, Kia; Mendoza, Christopher J; Norato, Gina; Cheung, Ken; Riekhof, Willa; Varma, Pooja; Colina-Prisco, Claudia; Emile-Backer, Magalie; Meilleur, Katherine G; Marks, Andrew R; Webb, Yael; Marcantonio, Eugene E; Foley, A Reghan; Bönnemann, Carsten G; Mohassel, Payam

Development of a proxy-reported scale to assess motor function in infants and young children with early-onset neuromuscular disorders

开发一种由代理人报告的量表,用于评估患有早期神经肌肉疾病的婴幼儿的运动功能

Capella-Peris, Carlos; Emile-Backer, Magalie; Shelton, Monique O; Chrismer, Irene C; Cosgrove, Mary M; Bendixen, Roxanna M; Meilleur, Katherine G

Randomized controlled trial of N-acetylcysteine therapy for RYR1-related myopathies

N-乙酰半胱氨酸治疗RYR1相关肌病的随机对照试验

Todd, Joshua J; Lawal, Tokunbor A; Witherspoon, Jessica W; Chrismer, Irene C; Razaqyar, Muslima S; Punjabi, Monal; Elliott, Jeffrey S; Tounkara, Fatoumata; Kuo, Anna; Shelton, Monique O; Allen, Carolyn; Cosgrove, Mary M; Linton, Melody; Michael, Darren; Jain, Minal S; Waite, Melissa; Drinkard, Bart; Wakim, Paul G; Dowling, James J; Bönnemann, Carsten G; Emile-Backer, Magalie; Meilleur, Katherine G

Mixed methods analysis of Health-Related Quality of Life in ambulant individuals affected with RYR1-related myopathies pre-post-N-acetylcysteine therapy

采用混合方法分析RYR1相关肌病患者在接受N-乙酰半胱氨酸治疗前后的健康相关生活质量。

Capella-Peris, Carlos; Cosgrove, Mary M; Chrismer, Irene C; Emile-Backer, Magalie; Razaqyar, M Sonia; Elliott, Jeffrey S; Kuo, Anna; Wakim, Paul G; Meilleur, Katherine G

Understanding Symptoms in RYR1-Related Myopathies: A Mixed-Methods Analysis Based on Participants' Experience

了解RYR1相关肌病症状:基于参与者经验的混合方法分析

Capella-Peris, Carlos; Cosgrove, Mary M; Chrismer, Irene C; Razaqyar, M Sonia; Elliott, Jeffrey S; Kuo, Anna; Emile-Backer, Magalie; Meilleur, Katherine G