Biomedicines · 15 authors, 15 centres
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The study found that while enzyme replacement therapy controlled visceral manifestations, neurological symptoms like psychomotor retardation generally worsened over time, highlighting disease heterogeneity and the limited efficacy of current treatment on neurological progression.
Results indicate that while ERT effectively controlled visceral manifestations like organomegaly and hematological issues, neurological symptoms such as psychomotor retardation progressed during follow-up. Severe skeletal manifestations like kyphosis also progressed, apparently unrelated to ERT.
