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Basil T. Darras

boston children's hospital
H指数:72
被引数:2.2万

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Intrathecal onasemnogene abeparvovec for treatment-experienced patients with spinal muscular atrophy: a phase 3b, open-label trial

鞘内注射onasemnogene abeparvovec治疗经治脊髓性肌萎缩症患者:一项3b期、开放标签试验

2025-12-080
OAAI
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
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
Analysis of human urinary extracellular vesicles reveals disordered renal metabolism in myotonic dystrophy type 1

对人类尿液外泌体的分析揭示了强直性肌营养不良1型中的肾代谢紊乱

2025-03-050
OA
Therapeutic Role of Nusinersen on Respiratory Progression in Pediatric Patients With Spinal Muscular Atrophy Type 2 and Nonambulant Type 3

Nusinersen对2型和3型非卧床型脊髓性肌萎缩症患儿呼吸进展的治疗作用

2024-06-014
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
Disease Trajectories in the Revised Hammersmith Scale in a Cohort of Untreated Patients with Spinal Muscular Atrophy types 2 and 3

未经治疗的2型和3型脊髓性肌萎缩症患者队列中修订的Hammersmith量表中的疾病轨迹

2024-02-275
OAAI
Nusinersen Treatment of Children with Later-Onset Spinal Muscular Atrophy and Scoliosis Is Associated with Improvements or Stabilization of Motor Function

Nusinersen治疗晚发性脊髓性肌萎缩症和脊柱侧凸的儿童与运动功能的改善或稳定有关

2023-07-268
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