Smith, P.B.

disease patient pompe pompe disease late-onset pompe disease study enzyme replacement therapy baseline enzyme late-onset treatment abnormality replacement mass index therapy hypertrophy group echocardiogram center duration signi interval ejection echocardiographic fraction ejection fraction ficant analysis 0.99 alpha-glucosidase index university glycogen control acid alpha-glucosidase treatment arm 78 weeks range change safety block van der ploeg tachycardia enzyme replacement number infantile-onset pompe disease medicine ef ficacy pr interval abnormalities identi fied children wolff-parkinson-white syndrome adult variable kishnani bundle branch volume genetic hospital table placebo signi ficant syndrome van doorn p storage signi ficant change trial genet identi pediatr status parameter interquartile range echocardiographic abnormalities function 78- ficacy disease duration echocardiography