Long-term follow-up of 17 patients with childhood Pompe disease treated with enzyme replacement therapy.
Journal Of Inherited Metabolic Disease
van der Meijden, Jan C JC; Kruijshaar, Michelle E ME; Harlaar, Laurike L; Rizopoulos, Dimitris D; van der Beek, Nadine A M E NAME; van der Ploeg, Ans T AT