Search Result "GM1 gangliosidosis"


Research Article

AAV9-coGLB1 Improves Lysosomal Storage and Rescues Central NervousSystem Inflammation in a Mutant Mouse Model of GM1 Gangliosidosis

Journal: Current Gene Therapy
Volume: 22 Issue: 4 Year: 2022 Page: 352-365
Author(s):

Development and Medical Application of Unsaturated Carbaglycosylamine Glycosidase Inhibitors

Journal: Mini-Reviews in Medicinal Chemistry
Volume: 7 Issue: 7 Year: 2007 Page: 679-691
Author(s): Seiichiro Ogawa, Miki Kanto, Yoshiyuki Suzuki, Seiichiro Ogawa, Miki Kanto, Yoshiyuki Suzuki

Miglustat: Substrate Reduction Therapy for Lysosomal Storage Disorders Associated with Primary Central Nervous System Involvement

Journal: Recent Patents on CNS Drug Discovery (Discontinued)
Volume: 1 Issue: 1 Year: 2006 Page: 77-82
Author(s): Gregory M. Pastores

The Two Faces of Iminoalditols: Powerful Inhibitors Trigger Glycosidase Activation

Journal: Current Enzyme Inhibition
Volume: 8 Issue: 1 Year: 2012 Page: 47-99
Author(s): Tanja M. Wrodnigg, Arnold E. Stutz

Treatment of Lysosomal Storage Diseases: Recent Patents and Future Strategies

Journal: Recent Patents on Endocrine, Metabolic & Immune Drug Discovery
Volume: 8 Issue: 1 Year: 2014 Page: 9-25
Author(s): Saida Ortolano,Irene Vieitez,Carmen Navarro,Carlos Spuch

Review Article

Enzyme Replacement Therapy for Genetic Disorders Associated withEnzyme Deficiency

Journal: Current Medicinal Chemistry
Volume: 29 Issue: 3 Year: 2022 Page: 489-525
Author(s): Marialaura Marchetti,Serena Faggiano,Andrea Mozzarelli

Mini-Review Article

Clinical Development of Cell Therapies to Halt Lysosomal Storage Diseases:Results and Lessons Learned

Journal: Current Gene Therapy
Volume: 22 Issue: 3 Year: 2022 Page: 191-213
Author(s): Valeria Graceffa

Lysosomal Acid Phosphatase Biosynthesis and Dysfunction: A Mini Review Focused on Lysosomal Enzyme Dysfunction in Brain

Journal: Current Molecular Medicine
Volume: 16 Issue: 5 Year: 2016 Page: 439-446
Author(s): N. Ashtari,X. Jiao,M. Rahimi-Balaei,S. Amiri,S. E. Mehr,B. Yeganeh,H. Marzban

Review Article

New Advanced Strategies for the Treatment of Lysosomal Diseases Affecting the Central Nervous System

Journal: Current Pharmaceutical Design
Volume: 25 Issue: 17 Year: 2019 Page: 1933-1950
Author(s): Maria R. Gigliobianco,Piera Di Martino,Siyuan Deng,Cristina Casadidio,Roberta Censi

Hydrops Fetalis and Fetal Alloimmune Thrombocytopenia

Ebook: Diagnosis and Management of Fetal Disorders
Volume: 1 Year: 2023
Author(s):
Doi: 10.2174/9789815079630123010012

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