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GM2 Gangliosidoses (GM2 Gangliosidosis)

A group of recessively inherited diseases characterized by the intralysosomal accumulation of G(M2) GANGLIOSIDE in the neuronal cells. Subtypes include mutations of enzymes in the BETA-N-ACETYLHEXOSAMINIDASES system or G(M2) ACTIVATOR PROTEIN leading to disruption of normal degradation of GANGLIOSIDES, a subclass of ACIDIC GLYCOSPHINGOLIPIDS.
Also Known As:
GM2 Gangliosidosis; Gangliosidoses GM2; Gangliosidoses, GM2; GM2 Gangliosidose; GM2, Gangliosidoses; Gangliosidose, GM2; Gangliosidosis, GM2; G(M2) Gangliosidoses
Networked: 267 relevant articles (10 outcomes, 21 trials/studies)

Relationship Network

Disease Context: Research Results

Related Diseases

1. Neurodegenerative Diseases (Neurodegenerative Disease)
2. Atrophy
3. Hypokinesia (Bradykinesia)
4. Sandhoff Disease (Sandhoff's Disease)
5. Type C Niemann-Pick Disease (Niemann Pick Disease, Type C)

Experts

1. Tsuji, Daisuke: 9 articles (09/2016 - 08/2006)
2. Itoh, Kohji: 8 articles (09/2016 - 08/2006)
3. Sakuraba, Hitoshi: 8 articles (09/2016 - 01/2002)
4. Walia, Jagdeep S: 6 articles (01/2022 - 03/2015)
5. Cox, Timothy M: 6 articles (11/2021 - 06/2009)
6. Platt, Frances M: 6 articles (01/2021 - 08/2002)
7. Martin, Douglas R: 6 articles (01/2020 - 11/2005)
8. Sandhoff, Konrad: 6 articles (01/2019 - 08/2002)
9. Martino, Sabata: 5 articles (06/2022 - 06/2002)
10. Gray, Steven J: 5 articles (01/2022 - 01/2016)

Drugs and Biologics

Drugs and Important Biological Agents (IBA) related to GM2 Gangliosidoses:
1. miglustat (Zavesca)FDA Link
2. Hexosaminidase BIBA
3. Biomarkers (Surrogate Marker)IBA
4. Pyrimethamine (Daraprim)FDA Link
5. Arginine (L-Arginine)FDA Link
6. Ursodeoxycholic Acid (Urso)FDA LinkGeneric
7. Peptide Hydrolases (Proteases)FDA Link
8. Levodopa (L Dopa)FDA LinkGeneric
9. Hexosaminidase A (Hex A)IBA
10. Hexosaminidases (Hexosaminidase)IBA

Therapies and Procedures

1. Therapeutics
2. Investigational Therapies (Experimental Therapy)
3. Enzyme Replacement Therapy
4. Off-Label Use
5. Cell- and Tissue-Based Therapy (Cell Therapy)