Efficacy of enzyme replacement therapy over six-months in a non-classic infantile Pompe disease form: our experience in a 2-year-old affected child

Efficacy of enzyme replacement therapy over six-months in a non-classic infantile Pompe disease form: our experience in a 2-year-old affected child / S. Paci, V. Scelsa, M. Gasparri, E. Salvatici. - In: JOURNAL OF INHERITED METABOLIC DISEASE. - ISSN 0141-8955. - 36:suppl. 2(2013 Sep), pp. 342-342. ( 12. ICIEM Barcelona 2013).