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Lysosomal Storage Diseases (Lysosomal Storage Disease)

Inborn errors of metabolism characterized by defects in specific lysosomal hydrolases and resulting in intracellular accumulation of unmetabolized substrates.
Also Known As:
Lysosomal Storage Disease; Disease, Lysosomal Storage; Diseases, Lysosomal Storage; Disorder, Lysosomal Enzyme; Disorders, Lysosomal Enzyme; Enzyme Disorder, Lysosomal; Enzyme Disorders, Lysosomal; Lysosomal Enzyme Disorder; Lysosomal Enzyme Disorders
Networked: 2132 relevant articles (48 outcomes, 89 trials/studies)

Relationship Network

Disease Context: Research Results

Related Diseases

1. Mucopolysaccharidoses
2. Mucopolysaccharidosis I (Hurler Syndrome)
3. Central Nervous System Diseases (CNS Diseases)
4. Neoplasms (Cancer)
5. Mucopolysaccharidosis VI (Syndrome, Maroteaux-Lamy)

Experts

1. Sands, Mark S: 27 articles (01/2021 - 12/2002)
2. Gieselmann, Volkmar: 24 articles (07/2022 - 12/2002)
3. Giugliani, Roberto: 23 articles (01/2022 - 04/2004)
4. Grabowski, Gregory A: 21 articles (05/2020 - 01/2003)
5. Platt, Frances M: 17 articles (01/2022 - 05/2004)
6. Matzner, Ulrich: 17 articles (01/2021 - 12/2002)
7. Vogler, Carole: 17 articles (10/2012 - 01/2003)
8. d'Azzo, Alessandra: 16 articles (03/2021 - 07/2005)
9. Tomatsu, Shunji: 16 articles (03/2020 - 05/2003)
10. Sly, William S: 16 articles (03/2016 - 03/2003)

Drugs and Biologics

Drugs and Important Biological Agents (IBA) related to Lysosomal Storage Diseases:
1. EnzymesIBA
2. Lysergic Acid Diethylamide (LSD)IBA
3. Proteins (Proteins, Gene)FDA Link
4. GlycosphingolipidsIBA
5. HydrolasesIBA
6. Iduronidase (alpha-L-Iduronidase)IBA
7. OligosaccharidesIBA
8. Amyloid (Amyloid Fibrils)IBA
9. Biomarkers (Surrogate Marker)IBA
10. Glucosylceramidase (Glucocerebrosidase)IBA

Therapies and Procedures

1. Therapeutics
2. Enzyme Replacement Therapy
3. Bone Marrow Transplantation (Transplantation, Bone Marrow)
4. Cell- and Tissue-Based Therapy (Cell Therapy)
5. Investigational Therapies (Experimental Therapy)