Byrne, B.J.

patient disease pompe pompe disease symptom onset registry month van der ploeg symptom onset cardiomyopathy enzyme replacement therapy diagnosis late-onset pompe disease muscle ≤12 pompe registry genzyme corporation enzyme kishnani alglucosidase alfa death byrne muscle weakness course therapy ploeg treatment van doorn late-onset genetic mutation weakness children disease course assessment alglucosidase pompe registry board genzyme study registry data glycogen percent cause reuser adult corporation history trial method analysis replacement follow-up table understanding metabolism fi ndings board ficiency function blood infantile-onset pompe disease fi rst year population pompe patients van den hout storage physician corzo 1–11 group enzyme assay hageman doorn baseline spectrum neurol outcome mandel support