pyrimethamine has been researched along with Gangliosidoses, GM2 in 3 studies
Maloprim: contains above 2 cpds
Gangliosidoses, GM2: 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.
| Excerpt | Relevance | Reference |
|---|---|---|
| "GM2 gangliosidoses are a group of pathologies characterized by GM2 ganglioside accumulation into the lysosome due to mutations on the genes encoding for the β-hexosaminidases subunits or the GM2 activator protein." | 5.05 | GM2 Gangliosidoses: Clinical Features, Pathophysiological Aspects, and Current Therapies. ( Alméciga-Díaz, CJ; Benincore-Flórez, E; Echeverri-Peña, OY; Espejo-Mojica, AJ; Garzón Jaramillo, RG; Leal, AF; Solano-Galarza, D; Suarez, DA, 2020) |
| "Late-onset GM2 gangliosidosis is an autosomal recessive, neurodegenerative, lysosomal storage disease, caused by deficiency of ß-hexosaminidase A (Hex A), resulting from mutations in the HEXA (Tay-Sachs variant) or the HEXB (Sandhoff variant) genes." | 2.76 | An open-label Phase I/II clinical trial of pyrimethamine for the treatment of patients affected with chronic GM2 gangliosidosis (Tay-Sachs or Sandhoff variants). ( Clarke, JT; Kolodny, EH; Mahuran, DJ; Raiman, JA; Rigat, BA; Sathe, S; Tropak, MB, 2011) |
| "Late-onset GM2 gangliosidosis is composed of two related, autosomal recessive, neurodegenerative diseases, both resulting from deficiency of lysosomal, heterodimeric beta-hexosaminidase A (Hex A, alphabeta)." | 1.34 | Pyrimethamine as a potential pharmacological chaperone for late-onset forms of GM2 gangliosidosis. ( Buttner, J; Clarke, JT; Kok, F; Maegawa, GH; Mahuran, DJ; Stockley, T; Tropak, M, 2007) |
| Timeframe | Studies, this research(%) | All Research% |
|---|---|---|
| pre-1990 | 0 (0.00) | 18.7374 |
| 1990's | 0 (0.00) | 18.2507 |
| 2000's | 1 (33.33) | 29.6817 |
| 2010's | 1 (33.33) | 24.3611 |
| 2020's | 1 (33.33) | 2.80 |
| Authors | Studies |
|---|---|
| Maegawa, GH | 1 |
| Tropak, M | 1 |
| Buttner, J | 1 |
| Stockley, T | 1 |
| Kok, F | 1 |
| Clarke, JT | 2 |
| Mahuran, DJ | 2 |
| Leal, AF | 1 |
| Benincore-Flórez, E | 1 |
| Solano-Galarza, D | 1 |
| Garzón Jaramillo, RG | 1 |
| Echeverri-Peña, OY | 1 |
| Suarez, DA | 1 |
| Alméciga-Díaz, CJ | 1 |
| Espejo-Mojica, AJ | 1 |
| Sathe, S | 1 |
| Kolodny, EH | 1 |
| Rigat, BA | 1 |
| Raiman, JA | 1 |
| Tropak, MB | 1 |
| Trial | Phase | Enrollment | Study Type | Start Date | Status | ||
|---|---|---|---|---|---|---|---|
| A Dose-Escalated, Double-Blind, Placebo-Controlled, Randomized Phase I Clinical Trial of Pyrimethamine in Patients Affected With Chronic GM2 Gangliosidosis (Tay-Sachs or Sandhoff Variants)[NCT00679744] | Phase 1 | 0 participants (Actual) | Interventional | 2008-05-31 | Withdrawn (stopped due to Due to lack of funding) | ||
| [information is prepared from clinicaltrials.gov, extracted Sep-2024] | |||||||
1 review available for pyrimethamine and Gangliosidoses, GM2
| Article | Year |
|---|---|
GM2 Gangliosidoses: Clinical Features, Pathophysiological Aspects, and Current Therapies.
Topics: 1-Deoxynojirimycin; beta-N-Acetylhexosaminidases; Blood-Brain Barrier; Clinical Trials as Topic; Die | 2020 |
1 trial available for pyrimethamine and Gangliosidoses, GM2
| Article | Year |
|---|---|
An open-label Phase I/II clinical trial of pyrimethamine for the treatment of patients affected with chronic GM2 gangliosidosis (Tay-Sachs or Sandhoff variants).
Topics: Adult; beta-Galactosidase; Enzyme Assays; Female; Gangliosidoses, GM2; Glucosylceramidase; Hexosamin | 2011 |
1 other study available for pyrimethamine and Gangliosidoses, GM2
| Article | Year |
|---|---|
Pyrimethamine as a potential pharmacological chaperone for late-onset forms of GM2 gangliosidosis.
Topics: beta-N-Acetylhexosaminidases; Dimerization; Dose-Response Relationship, Drug; Enzyme Inhibitors; Fib | 2007 |