Long-term galsulfase enzyme replacement therapy in Taiwanese mucopolysaccharidosis VI patients: A case series
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Abbreviations
MPS
mucopolysaccharidosis
ASB
N-acetylgalactosamine 4-sulfatase
GAG
glycosaminoglycan
ERT
enzyme replacement therapy
Galsulfase
recombinant human N-acetylgalactosamine 4-sulfatase
Z score
standard deviation score
6MWT
6-minute walk test
3MSCT
3-minute stair climb test
FVC
Forced vital capacity
FEV1
forced expiratory volume in 1 s
E/A
ratio between early and late (atrial) ventricular filling velocity
LVMI
left ventricular mass index
IVSd
interventricular septum thickness in diastole
LVPWd
left ventricular posterior wall thickness in diastole
LVM
left ventricular mass
BMD
bone mineral density
HAZ
height-for-age
DXA
dual energy x-ray absorptiometry
PTA
pure-tone audiometry
AC
air conduction
BC
bone conduction
HAQ
Health Assessment Questionnaire
CHAQ
Childhood Health Assessment Questionnaire
Keywords
Cardiac hypertrophy
Diastolic dysfunction
Enzyme replacement therapy
Glycosaminoglycans
Mucopolysaccharidosis VI
Pulmonary function
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© 2016 The Authors. Published by Elsevier Inc.