Japan ElapraseŽ Treatment (JET) study: Idursulfase enzyme replacement therapy in adult patients with attenuated Hunter syndrome (Mucopolysaccharidosis II, MPS …

T Okuyama, A Tanaka, Y Suzuki, H Ida… - Molecular genetics and …, 2010 - Elsevier
This open-label clinical study enrolled 10 adults with attenuated Mucopolysaccharidosis II
and advanced disease under the direction of the Japan Society for Research on
Mucopolysaccharidosis Disorders prior to regulatory approval of idursulfase in Japan. Ten
male patients, ages 21–53years, received weekly intravenous infusions of 0.5 mg/kg
idursulfase for 12months. Significant reductions in lysosomal storage and several clinical
improvements were observed during the study (mean changes below). Urinary …