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Journal of Pediatrics, The, ISSN 0022-3476, 2015, Volume 166, Issue 4, pp. 1075 - 1078.e1
Pompe disease (PD), or glycogen storage disease type II, results from deficiency of acid α-glucosidase. Patients with infantile-onset PD die by early childhood... 
Pediatrics | PEDIATRICS | ADRENARCHE | THERAPY | GIRLS | ONSET | Puberty | Prognosis | Follow-Up Studies | Humans | Child, Preschool | Infant | Male | Glycogen Storage Disease Type II - therapy | Glycogen Storage Disease Type II - physiopathology | Enzyme Replacement Therapy - methods | Sexual Development | Female | Retrospective Studies | Child | Enzymes | Neurosciences | Ophthalmology | Glycogen | Medical genetics
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