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Thomas O. Crawford

Johns Hopkins Hospital
H指数:78
被引数:2.1万

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New SMA era: A broad-range tiered assessment of function for the evolving SMA phenotype (EVOLVE-SMA)

新SMA时代:针对不断演变的SMA表型的广谱分级功能评估(EVOLVE-SMA)

2025-12-300
PRE
A plain language summary of the SAPPHIRE clinical trial of apitegromab in children and young adults with spinal muscular atrophy

SAPPHIRE临床试验的简明语言摘要,该试验研究apitegromab在儿童和年轻成人脊髓性肌萎缩症患者中的应用。

2025-11-130
OAAI
A prospective, multi-center, observational study of the safety, tolerability and effectiveness of nusinersen in adult patients with Spinal Muscular Atrophy

一项关于nusinersen在成人脊髓性肌萎缩症患者中安全性、耐受性和有效性的前瞻性、多中心、观察性研究。

2025-10-240
PRE
Consensus recommendations and considerations for the delivery and monitoring of gene therapy in patients with Duchenne muscular dystrophy

关于在杜氏肌营养不良症患者中实施基因治疗及监测的共识推荐与考量

2025-08-230
PRE
Safety and efficacy of apitegromab in nonambulatory type 2 or type 3 spinal muscular atrophy (SAPPHIRE): a phase 3, double-blind, randomised, placebo-controlled trial

阿皮特格罗玛ab在非行走型2型或3型脊髓性肌萎缩症中的安全性与有效性(SAPPHIRE研究):一项III期、双盲、随机、安慰剂对照试验

2025-08-130
PRE
Combined clinical, structural and cellular studies discriminate pathogenic and benign TRPV4 variants
BRAIN
IF11.7
2024-07-181
PRE
TRPV4 neuromuscular disease registry highlights bulbar, skeletal and proximal limb manifestations

TRPV4神经肌肉疾病注册表突出了球部,骨骼和近端肢体表现

BRAIN
IF11.7
2024-06-251
PRE
Intrathecal Gene Therapy for Giant Axonal Neuropathy
2024-03-219
PRE
Safety and Efficacy of Apitegromab in Patients With Spinal Muscular Atrophy Types 2 and 3

Apitegromab在2型和3型脊髓性肌萎缩症患者中的安全性和有效性

2024-03-126
OAAI
Intrathecal Onasemnogene Abeparvovec for Sitting, Nonambulatory Patients with Spinal Muscular Atrophy: Phase I Ascending-Dose Study (STRONG)

鞘内注射Onasemnogene Abeparvovec用于坐着,非卧床的脊髓性肌萎缩症患者: I期递增剂量研究 (STRONG)

2023-05-0221
OAAI
Mapping gas around massive galaxies: cross-correlation of DES Y3 galaxies and Compton-y maps from SPT and Planck

映射大质量星系周围的气体: SPT和Planck的DES Y3星系和compton-y图的互相关

2023-04-205
OA
Intron mutations and early transcription termination in Duchenne and Becker muscular dystrophy

杜氏和贝克尔肌营养不良症的内含子突变和早期转录终止

2022-03-0727
OA
Clinical and genetic spectrum of a large cohort of patients with δ-sarcoglycan muscular dystrophy
BRAIN
IF11.7
2021-09-1316
OAAI