Effect of cyclic, low dose pyrimethamine treatment in patients with Late Onset Tay Sachs: an open label, extended pilot study

BACKGROUND: Late Onset Tay- Sachs disease (LOTS) is a rare neurodegenerative lysosomal storage disease which results from mutations in the gene encoding the α subunit (HEXA) of β-hexosaminidase enzyme (HexA). At the present time, no effective treatment exists for LOTS and other neurodegenerative dis...

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Detalles Bibliográficos
Autores principales: Osher, Etty, Fattal-Valevski, Aviva, Sagie, Liora, Urshanski, Nataly, Sagiv, Nadav, Peleg, Leah, Lerman-Sagie, Tally, Zimran, Ari, Elstein, Deborah, Navon, Ruth, Valevski, Avi, Stern, Naftali
Formato: Online Artículo Texto
Lenguaje:English
Publicado: BioMed Central 2015
Materias:
Research
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4404274/
https://www.ncbi.nlm.nih.gov/pubmed/25896637
http://dx.doi.org/10.1186/s13023-015-0260-7

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4404274/
https://www.ncbi.nlm.nih.gov/pubmed/25896637
http://dx.doi.org/10.1186/s13023-015-0260-7

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