Enzyme replacement therapy: improving outcomes in rare disease
European Pharmaceutical Review
SEPTEMBER 12, 2024
ASMD is a rare disorder affecting the breakdown of certain complex fats in the body,” explained Dr Robin Lachmann, Principal Investigator at the National Hospital for Neurology and Neurosurgery, following the dosing of the first patient in a Sanofi Genzyme Phase II/III trial of olipudase alfa. What is working well and what is lacking?
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