and post-hoc data reveal SMA Type 1 patients could speak, swallow and maintain airway protection. Retrieved March 14, 2022, from https://www.novartis.com/news/media-releases/novartis-data-again-demonstrate-age-appropriate-development-when-zolgensma-used-presymptomatically-and-post-...
[1].Servais L, Baranello G, Boespflug-Tanguy O, et al. P.109 FIREFISH Parts 1 and 2: 36-month safety and efficacy of risdiplam in Type 1 spinal muscular atrophy (SMA). Presented at the 27th International Annual Congress of the World Muscle Society, Halifax, Canada, October 11-15, ...
[2]. Hua Y, Sahashi K, Hung G, Rigo F, Passini MA, Bennett CF, Krainer AR. Antisense correction of SMN2 splicing in the CNS rescues necrosis in a type III SMA mouse model. Genes Dev. 2010 Aug 1; 24(15):16344-44. [3]. Finkel R, Chiriboga C, Vajsar J, et al. Treatment ...
5q-SMA是SMA的最常见类型,约占全部SMA病例的95%,该类型SMA是由5号染色体上的SMN1(运动神经元生存蛋白1)基因突变所引起的,因此得名5q-SMA。 原文出处:Roche’s Risdiplam meets primary endpoint in pivotal FIREFISH trial in infants with type 1 spinal muscular atrophy...
RF射频连接器同轴线连接器布尔金bulgin通用射频连接器特价荐 规格 1.85mm系列、2.4mm系列、2.92mm系列、N,Type系列、SMA系列、SSMA系列、TNCA系列、RF适配系列、射频终端系列 深圳市益德科技有限公司10年 月均发货速度:暂无记录 广东 深圳市宝安区 ¥1.05成交70条 ...
[1] Dabbous, O., et al., Survival, Motor Function, and Motor Milestones: Comparison of AVXS-101 Relative to Nusinersen for the Treatment of Infants with Spinal Muscular Atrophy Type 1. Adv Ther, 2019. 36(5): p. 1164-1176. [2]Available from: ...
[1] Mohseni, R., Hamidieh, A.A., Shoae-Hassani, A. et al. An open-label phase 1 clinical trial of the allogeneic side population adipose-derived mesenchymal stem cells in SMA type 1 patients. Neurol Sci (2021). https://doi.org/10.1007/s10072-021-05291-2 ...
Who: 121 children aged 7 months and younger with Type 1 (early-onset) SMA Study time: 13 months Primary outcomes: Time to death or use of permanent assisted ventilation The proportion of responders (or number of children) who showed an improvement in motor milestones according to HINE-2 Sa...
[1] Single-Dose Gene-Replacement Therapy for Spinal Muscular Atrophy PMID: 29091557 DOI: 10.1056/NEJMoa1706198 [2] Mendell JR, Al Zaidy S, Shell R., et al. AVXS-101 Phase 1 Gene Replacement Therapy Clinical Trial in SMA Type 1: Event-Free Survival and Achievement of Developmental Milestone...
[1] Dabbous, O., et al., Survival, Motor Function, and Motor Milestones: Comparison of AVXS-101 Relative to Nusinersen for the Treatment of Infants with Spinal Muscular Atrophy Type 1. Adv Ther, 2019. 36(5): p. ...