Long-term benefit of enzyme replacement therapy in Pompe disease: A 5-year prospective study.

Kuperus, Esther

Long-term benefit of enzyme replacement therapy in Pompe disease: A 5-year prospective study. [electronic resource] - Neurology Dec 2017 - 2365-2373 p. digital

Publication Type: Journal Article

1526-632X

10.1212/WNL.0000000000004711 doi


Activities of Daily Living
Adult
Aged
Cohort Studies
Enzyme Replacement Therapy--adverse effects
Female
Follow-Up Studies
Glycogen Storage Disease Type II--drug therapy
Humans
Male
Middle Aged
Muscle Strength
Muscle Strength Dynamometer
Prospective Studies
Respiratory Function Tests
Treatment Outcome
Walk Test
Young Adult
alpha-Glucosidases--therapeutic use