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

Validation of high-sensitivity assays to quantitate cerebrospinal fluid and serum β-galactosidase activity in patients with GM1-gangliosidosis.

验证高灵敏度检测方法在GM1神经节苷脂病患者脑脊液和血清β-半乳糖苷酶活性定量中的应用

Quadrini Karen J, Vrentas Catherine, Duke Christian, Wilson Chris, Hinderer Christian J, Weinstein David A, Al-Zaidy Samiah A, Browne Susan E, Wilson James M, Ni Yan G

Five-Year Extension Results of the Phase 1 START Trial of Onasemnogene Abeparvovec in Spinal Muscular Atrophy

Onasemnogene Abeparvovec治疗脊髓性肌萎缩症的1期START试验五年扩展结果

Mendell, Jerry R; Al-Zaidy, Samiah A; Lehman, Kelly J; McColly, Markus; Lowes, Linda P; Alfano, Lindsay N; Reash, Natalie F; Iammarino, Megan A; Church, Kathleen R; Kleyn, Aaron; Meriggioli, Matthew N; Shell, Richard

Gene Delivery for Limb-Girdle Muscular Dystrophy Type 2D by Isolated Limb Infusion

通过肢体输注进行肢带型肌营养不良症2D型基因递送

Mendell, Jerry R; Chicoine, Louis G; Al-Zaidy, Samiah A; Sahenk, Zarife; Lehman, Kelly; Lowes, Linda; Miller, Natalie; Alfano, Lindsay; Galliers, Beverly; Lewis, Sarah; Murrey, Darren; Peterson, Ellyn; Griffin, Danielle A; Church, Kathleen; Cheatham, Sharon; Cheatham, John; Hogan, Mark J; Rodino-Klapac, Louise R

A Roadmap to Newborn Screening for Duchenne Muscular Dystrophy

新生儿杜氏肌营养不良症筛查路线图

Al-Zaidy, Samiah A; Lloyd-Puryear, Michele; Kennedy, Annie; Lopez, Veronica; Mendell, Jerry R

Follistatin Gene Therapy Improves Ambulation in Becker Muscular Dystrophy

卵泡抑素基因疗法改善贝克尔型肌营养不良症患者的行走能力

Al-Zaidy, Samiah A; Sahenk, Zarife; Rodino-Klapac, Louise R; Kaspar, Brian; Mendell, Jerry R