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

Endocannabinoid receptor 2 is a potential biomarker and therapeutic target for the lysosomal storage disorders

内源性大麻素受体 2 是溶酶体贮积症的潜在生物标志物和治疗靶点

Calogera M Simonaro, Makiko Yasuda, Edward H Schuchman

Acid Ceramidase Protects Against Hepatic Ischemia/Reperfusion Injury by Modulating Sphingolipid Metabolism and Reducing Inflammation and Oxidative Stress

酸性神经酰胺酶通过调节鞘脂代谢、减少炎症和氧化应激来保护肝脏免受缺血/再灌注损伤。

Jiang, Yuan; He, Xingxuan; Simonaro, Calogera M; Yi, Bin; Schuchman, Edward H

New paradigms for the treatment of lysosomal storage diseases: targeting the endocannabinoid system as a therapeutic strategy

溶酶体贮积症治疗的新范式:以内源性大麻素系统为靶点的治疗策略

Schuchman, Edward H; Ledesma, Maria D; Simonaro, Calogera M

Safety Study of Sodium Pentosan Polysulfate for Adult Patients with Mucopolysaccharidosis Type II

戊聚糖多硫酸钠治疗成人II型粘多糖贮积症患者的安全性研究

Orii, Kenji; Lim, Alaena; Tomatsu, Shunji; Stapleton, Molly; Suzuki, Yasuyuki; Simonaro, Calogera M; Schuchman, Edward H; Fukao, Toshiyuki; Matsumoto, Tadashi

Pentosan Polysulfate Treatment of Mucopolysaccharidosis Type IIIA Mice

戊聚糖多硫酸盐治疗粘多糖贮积症 IIIA 型小鼠

Ningning Guo, Victor DeAngelis, Changzhi Zhu, Edward H Schuchman, Calogera M Simonaro

Enzyme replacement therapy for Farber disease: Proof-of-concept studies in cells and mice

法伯氏病的酶替代疗法:细胞和小鼠的概念验证研究

Xingxuan He, Shaalee Dworski, Changzhi Zhu, Victor DeAngelis, Alex Solyom, Jeffrey A Medin, Calogera M Simonaro, Edward H Schuchman

Pentosan Polysulfate: Oral Versus Subcutaneous Injection in Mucopolysaccharidosis Type I Dogs

戊聚糖多硫酸盐:I 型粘多糖贮积症犬口服与皮下注射治疗

Calogera M Simonaro, Shunji Tomatsu, Tracy Sikora, Francyne Kubaski, Michael Frohbergh, Johana M Guevara, Raymond Y Wang, Moin Vera, Jennifer L Kang, Lachlan J Smith, Edward H Schuchman, Mark E Haskins

Intra-articular enzyme replacement therapy with rhIDUA is safe, well-tolerated, and reduces articular GAG storage in the canine model of mucopolysaccharidosis type I

在犬I型粘多糖贮积症模型中,关节内注射rhIDUA酶替代疗法安全、耐受性良好,并能减少关节内糖胺聚糖(GAG)的沉积。

Wang, Raymond Y; Aminian, Afshin; McEntee, Michael F; Kan, Shih-Hsin; Simonaro, Calogera M; Lamanna, William C; Lawrence, Roger; Ellinwood, N Matthew; Guerra, Catalina; Le, Steven Q; Dickson, Patricia I; Esko, Jeffrey D

Dose responsive effects of subcutaneous pentosan polysulfate injection in mucopolysaccharidosis type VI rats and comparison to oral treatment

皮下注射戊聚糖多硫酸盐对 VI 型粘多糖贮积症大鼠的剂量反应效应及与口服治疗的比较

Michael Frohbergh, Yi Ge, Fanli Meng, Nesrin Karabul, Alexander Solyom, Alon Lai, James Iatridis, Edward H Schuchman, Calogera M Simonaro

Systemic ceramide accumulation leads to severe and varied pathological consequences

全身性神经酰胺蓄积会导致严重且多样的病理后果。

Alayoubi, Abdulfatah M; Wang, James C M; Au, Bryan C Y; Carpentier, Stéphane; Garcia, Virginie; Dworski, Shaalee; El-Ghamrasni, Samah; Kirouac, Kevin N; Exertier, Mathilde J; Xiong, Zi Jian; Privé, Gilbert G; Simonaro, Calogera M; Casas, Josefina; Fabrias, Gemma; Schuchman, Edward H; Turner, Patricia V; Hakem, Razqallah; Levade, Thierry; Medin, Jeffrey A