Enzyme Replacement Trial Ended Early After Failing to Meet Goals

Enzyme Replacement Trial Ended Early After Failing to Meet Goals

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Enzyme Replacement Trial Ended Early After Failing to Meet Goals

A clinical trial extension study involving 12 children with Sanfilippo syndrome type A was terminated early, after efficacy analyses found that the investigational enzyme replacement therapy it was testing failed to show evidence that it could slow the patients’ progressive cognitive or neurological impairment. After a median of 264.4 weeks — just over five years — the researchers concluded that the enzyme replace therapy (ERT) was “unable to slow the neurocognitive decline of patients.” “This study…

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