D-galactose supplementation in individuals with PMM2-CDG: results of a multicenter, open label, prospective pilot clinical trial

PMM2-CDG is the most prevalent congenital disorder of glycosylation (CDG) with only symptomatic therapy. Some CDG have been successfully treated with D-galactose. We performed an open-label pilot trial with D-galactose in 9 PMM2-CDG patients. Overall, there was no significant improvement but some mi...

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Detalles Bibliográficos
Autores principales: Witters, Peter, Andersson, Hans, Jaeken, Jaak, Tseng, Laura, van Karnebeek, Clara D. M., Lefeber, Dirk J., Cassiman, David, Morava, Eva
Formato: Online Artículo Texto
Lenguaje:English
Publicado: BioMed Central 2021
Materias:
Letter to the Editor
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7980351/
https://www.ncbi.nlm.nih.gov/pubmed/33743737
http://dx.doi.org/10.1186/s13023-020-01609-z

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