International best practice for the evaluation of responsiveness to sapropterin dihydrochloride in patients with phenylketonuria.
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DOI: 10.1016/j.ymgme.2019.04.004
Abstract: Phenylketonuria (PKU) is an inherited metabolic disease caused by phenylalanine hydroxylase (PAH) deficiency. As the resulting high blood phenylalanine (Phe) concentration can have detrimental effects on brain development and function, international guidelines recommend lifelong control… read more here.
Keywords: responsiveness; sapropterin dihydrochloride; blood phe; phe ... See more keywords
PP14 Budget Impact Of Sapropterin Dihydrochloride For Phenylketonuria
Sign Up to like & getrecommendations! Published in 2019 at "International Journal of Technology Assessment in Health Care"
DOI: 10.1017/s0266462319001880
Abstract: Introduction The National Committee for Health Technology Incorporation (CONITEC) evaluates health technologies to recommend their inclusion or exclusion within the Brazilian Public Health System (SUS), and uses the budget impact assessment to estimate costs to… read more here.
Keywords: sapropterin dihydrochloride; budget impact; impact; health ... See more keywords