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Autor:
D Marravalle, Amf Garzone, V Moretti, A Mannucci, E Andresciani, A Ficcadenti, De Meo, F Ciuccarelli, A Pompilio
Publikováno v:
European Journal of Hospital Pharmacy. 21:A19.2-A19
Background There are almost 8,000 rare diseases (RD) (80% are genetically based) and 50% of them affect children. They are often highly complex metabolic diseases (MD): factors include heterogeneity (age of occurrence, aetiopathogenesis and symptomat