Results 91 to 100 of about 7,599 (237)

New era in treatment for phenylketonuria: Pharmacologic therapy with sapropterin dihydrochloride [PDF]

open access: yes, 2010
Oral administration of sapropterin hydrochloride, recently approved for use by the US Food and Drug Administration and the European Commission, is a novel approach for the treatment of phenylketonuria (PKU), one of the most common inborn errors of ...
Harding, Cary O
core   +2 more sources

Long-Term Treatment and Diagnosis of Tetrahydrobiopterin-Responsive Hyperphenylalaninemia with a Mutant Phenylalanine Hydroxylase Gene [PDF]

open access: bronze, 2003
Haruo Shintaku   +12 more
openalex   +1 more source

Complete correction of hyperphenylalaninemia following liver-directed, recombinant AAV2/8 vector-mediated gene therapy in murine phenylketonuria [PDF]

open access: green, 2005
Cary O. Harding   +6 more
openalex   +1 more source

897. Recombinant AAV2/8 Vector-Mediated Gene Therapy Corrects Hyperphenylalaninemia in Murine PKU [PDF]

open access: hybrid, 2005
Cary O. Harding   +4 more
openalex   +1 more source

More dat on sex ratio in hyperphenylalaninemia

open access: bronze, 1971
J.H. Easton   +2 more
openalex   +1 more source

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