Cognitive Outcome of Infants with Pompe Disease Receiving Enzyme-Replacement Therapy

Abstract

Researchers at University Medical Center, Rotterdam, the Netherlands, and University of Leuven, Belgium prospectively assessed cognitive function in 10 children with classic infantile Pompe disease who had been treated with enzyme-replacement therapy (ERT) since 1999.

Keywords

• severity of motor problems
• speech/language delay
• reliable assessment of cognition