Galsulfase (Naglazyme) therapy in infants with mucopolysaccharidosis VI.
Harmatz PR, Garcia P, Guffon N, Randolph LM, Shediac R, Braunlin E, Lachman RS, Decker C.
Harmatz PR, et al.
J Inherit Metab Dis. 2014 Mar;37(2):277-87. doi: 10.1007/s10545-013-9654-7. Epub 2013 Oct 10.
J Inherit Metab Dis. 2014.
PMID: 24108527
Free PMC article.
Clinical Trial.
RESULTS: Four infants (aged 3.3-12.7 months) participated in the study. Galsulfase was well tolerated at 1.0 and 2.0 mg/kg/week dose levels with no drug-related serious adverse events. ...Urinary GAG levels decreased with treatment. Early initiation of galsulfase ma …
RESULTS: Four infants (aged 3.3-12.7 months) participated in the study. Galsulfase was well tolerated at 1.0 and 2.0 mg/kg/week dose …