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Emma Ciafaloni

university of rochester medical center
H指数:49
被引数:8,635

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Study of Testosterone and Recombinant Human Growth Hormone in Facioscapulohumeral Muscular Dystrophy

研究睾酮和重组人生长激素在面肩肱型肌营养不良中的作用

2025-10-011
PRE
AAV gene therapy for Duchenne muscular dystrophy: the EMBARK phase 3 randomized trial

AAV基因治疗杜氏肌营养不良症: EMBARK 3期随机试验

2024-10-093
OAAI
Long-Term Follow-Up Cares and Check Initiative: A Program to Advance Long-Term Follow-Up in Newborns Identified with a Disease through Newborn Screening

长期随访护理和检查计划: 通过新生儿筛查确定患有疾病的新生儿的长期随访计划

2024-04-181
OAAI
Newborn screening for Duchenne muscular dystrophy: A two-year pilot study

Duchenne肌营养不良的新生儿筛查: 一项为期两年的初步研究

2023-06-2318
OA
Results from a 36-Week Open-Label Study of Recombinant Human Growth Hormone and Testosterone in Facioscapulohumeral Muscular Dystrophy (FSHD)
2023-04-250
PRE
Minimal manifestation status and prednisone withdrawal in the MGTX trial
2020-08-1115
OA
Implementation of population-based newborn screening reveals low incidence of spinal muscular atrophy

实施基于人群的新生儿筛查显示脊髓性肌萎缩症的发生率较低

2020-08-0152
OAAI