Hematopoietic stem cell transplantation in mucopolysaccharidosis type IIIA: A case description and comparison with a genotype-matched control group

BACKGROUND: Mucopolysaccharidosis type IIIA (MPS IIIA, Sanfilippo A syndrome) is a chronic progressive neurodegenerative storage disorder caused by a deficiency of lysosomal sulfamidase. The clinical hallmarks are sleep disturbances, behavioral abnormalities and loss of cognitive, speech and motor a...

Descripción completa

Detalles Bibliográficos
Autores principales: Köhn, Anja F., Grigull, Lorenz, du Moulin, Marcel, Kabisch, Sarah, Ammer, Luise, Rudolph, Cornelia, Muschol, Nicole M.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Elsevier 2020
Materias:
Research Paper
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7093801/
https://www.ncbi.nlm.nih.gov/pubmed/32226768
http://dx.doi.org/10.1016/j.ymgmr.2020.100578

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7093801/
https://www.ncbi.nlm.nih.gov/pubmed/32226768
http://dx.doi.org/10.1016/j.ymgmr.2020.100578

Ejemplares similares