Rare Disease Report

Enzyme Replacement Therapy and Morquio A - Predicting Outcomes

OCTOBER 11, 2015
James Radke, PhD



Paul Harmatz, MD of UCSF Benoits Children's Hospital Oakland talks about the difficulty in predicting outcomes for enzyme replacement therapy to treat patients with Morquio syndrome type A.

However, Dr Harmatz believes all patients with Morquio A should receive the treatment and to monitor outcomes both before and after treatment.

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