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Novel chemical and pharmacological therapies for PKU

Novel chemical and pharmacological therapies for PKU
  • Endocrinology and metabolism
  • Phenylketonuria (PKU)

Novel chemical and pharmacological therapies for PKU K. Michael Gibson (USA)

K. Michael Gibson (USA)

Dietary therapy in patients with phenylketonuria (PKU) represents the gold-standard of treatment, yet cognitive deficits persist in treated patients, over-restriction of amino acid intake may have untoward effects, and lifelong adherence to dietary restriction is challenging. We have begun to outline a small-molecule pharmacotherapeutic approach employing non-physiological amino acids(NPAAs) to lower brain phenylalanine (PHE) levels in the murine model of PKU Pahenu2 mice. Our objective is to maximally restrict PHE accretion into brain while minimally impacting other large neutral amino acids (LNAAs). Our novel chemico-pharmacological approach may provide new tools for PKU treatment that, even if it does not replace dietary restriction, could ultimately loosen dietary restriction in PKU patients.

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Target audience
Specialists in pediatric, Dietitians, nutritionists, clinical biochemists, genetists, basic scientists, psychologists, Nurses, Healthcare professionals
by Excemed
Endocrinology and metabolism