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Manisha Balwani

oregon health & science university
H指数:40
被引数:6,051

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Lipid profile in pediatric and adult patients with lysosomal acid lipase deficiency treated with sebelipase alfa: longitudinal evidence from the International LAL-D Registry

脂质谱在应用塞贝立泊治疗的患者(包括儿童和成人患者)中的变化:来自国际LAL-D登记研究的纵向证据

2026-08-010
PRE
Bitopertin Shows Efficacy in Patients with Erythropoietic Protoporphyria: Results from the Randomized, Double-Blind, Placebo-Controlled AURORA Trial

Bosipertin 在红细胞生成性原卟啉病患者中显示出疗效:来自随机、双盲、安慰剂对照的AURORA试验的结果

2025-12-110
OAAI
Long-term effectiveness of eliglustat treatment: A real-world analysis from the International Collaborative Gaucher Group Gaucher Registry

eliglustat治疗的长期有效性: 来自国际合作Gaucher Group Gaucher注册表的真实世界分析

2024-04-301
OAAI
Regression of smoldering myeloma with treatment of Gaucher disease
2024-03-272
OAAI
Evidence-based consensus guidelines for the diagnosis and management of erythropoietic protoporphyria and X-linked protoporphyria
2023-12-0114
OAAI
Evidence-based consensus guidelines for the diagnosis and management of protoporphyria-related liver dysfunction in erythropoietic protoporphyria and X-linked protoporphyria
2023-07-275
PRE