Study Results in Reducing Lysosomal Storage

Liver size decreased sharply by the first assessment at 6 weeks with 6 of 10 patients reaching a normal liver size. By 52 weeks, 9 of 10 patients had a normal liver size with a mean decrease of 25% in size. Spleen size also reduced rapidly, reaching a 20% reduction at 52 weeks. The reductions in liver and spleen size were maintained over 104 weeks. Urinary GAG excretion decreased rapidly within a few weeks of initiating infusions, reaching 63% reduction in 26 weeks, and 72% reduction in 52 weeks. By 104 weeks, the mean urinary GAG excretion was within the normal range for the group of patients, although levels in certain individual patients were still above normal. Overall, the study showed that lysosomal storage, as determined by organ size or urinary GAG excretion, rapidly declines with rhIDU infusions.

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