Experimental enzyme therapy tested for devastating childhood brain disease

This study tested an experimental enzyme replacement therapy called METAZYM in a single child with a severe, rare brain disease called late infantile MLD. The child had already received a stem cell transplant. Researchers gave intravenous enzyme infusions every other week for 18 months to see if it could help control nerve damage and improve function. The goal was to see if adding this enzyme therapy could help slow the progression of this currently untreatable disease.