Impact of 144 weeks of laronidase therapy on body functions, endurance and general well-being in a Hurler-Scheie patient.
The quality of life of a Hurler-Scheie patient who experienced improvement in several organ functions and regained mobility after 144 weeks of laronidase enzyme replacement therapy is described.
Soutar
R L
RL
Department of Haematology, Western Infirmary, Dumbarton Road, Glasgow, G11 6NT, UK, richard.soutar@northglasgow.scot.nhs.uk.
Mercer
J
J
Wraith
J E
JE
D000293:Adolescent; D005260:Female; D006801:Humans; D007068:Iduronidase / Q000008:administration & dosage / Q000627:therapeutic use*; D008059:Mucopolysaccharidosis I / Q000188:drug therapy* / Q000503:physiopathology; D011788:Quality of Life; D011994:Recombinant Proteins / Q000008:administration & dosage / Q000627:therapeutic use
D011994:Recombinant Proteins; D007068:Iduronidase