TITLE

Enzyme replacement therapy and/or hematopoietic stem cell transplantation at diagnosis in patients with mucopolysaccharidosis type I: results of a European consensus procedure

PUB. DATE
January 2011
SOURCE
Orphanet Journal of Rare Diseases;2011, Vol. 6 Issue 1, p55
SOURCE TYPE
Academic Journal
DOC. TYPE
Article
ABSTRACT
The article presents a European consensus procedure which showed the use of enzyme replacement therapy (ERT) and hematopoietic stem cell transplantation (HSCT) in the treatment of patient having mucopolysaccharidosis type I (MPS I). In this procedure a panel of specialists developed consensus-based statements on MPS I treatment. It informs that consensus procedure presented some issues which are related with therapeutic choices and research for MPS I.
ACCESSION #
67032165

 

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