Mucopolysaccharidosis I under enzyme replacement therapy with laronidase--a mortality case with autopsy report.
There is little information about MPS I-related complications during laronidase therapy. We describe the first autopsy report of a young male MPS I patient who died of infection-induced cardiopulmonary failure following 2 years of weekly treatment with laronidase.
Lin
H-Y
HY
Department of Pediatrics, Mackay Memorial Hospital, No. 92, Sec. 2, Chung-Shan North Road, Taipei 10449, Taiwan.
Lin
S-P
SP
Chuang
C-K
CK
Chen
M-R
MR
Chen
B-F
BF
Wraith
J E
JE
D000328:Adult; D001344:Autopsy; D017809:Fatal Outcome; D006025:Glycosaminoglycans / Q000378:metabolism; D006333:Heart Failure; D006497:Heparan Sulfate / Q000378:metabolism; D006801:Humans; D007068:Iduronidase / Q000737:chemistry / Q000172:deficiency / Q000627:therapeutic use*; D007239:Infections / Q000401:mortality; D008297:Male; D008059:Mucopolysaccharidosis I / Q000188:drug therapy* / Q000401:mortality*; D004364:Pharmaceutical Preparations; D011994:Recombinant Proteins / Q000627:therapeutic use
D006025:Glycosaminoglycans; D006497:Heparan Sulfate; D007068:Iduronidase; D004364:Pharmaceutical Preparations; D011994:Recombinant Proteins