APR targets the inherited metabolic disorders

APR is currently developing an innovative food for special medical purposes (Medical Food), intended to offer a new treatment option to Phenylketonuria (PKU), a rare, genetic, recessive metabolic disorder affecting about 50’000 people worldwide (on average 1:10’000 new born) and characterized by the lack or the malfunctioning of a liver enzyme needed to process phenylalanine (“PHE”), an essential amino acid.

If not properly treated, PKU leads to severe, non-recoverable mental retardation and major cognitive impairments. PKU can only be treated through a strict, life-long, low-protein (low-PHE) dietetic treatment combined with a daily assumption of Medical Food, which provides PHE-free amino acids and other important nutrients, though dietary compliance is still a key issue and, despite the diet, some neurological or psychosocial issues may occur.

APR developed and patented a technology to provide PKU patients with an innovative PHE-free Medical Food, engineered to allow a physiological absorption of amino acids, mimicking that of dietary proteins.

This advanced formulation has the potential of contributing to maintain PHE levels within the recommended ranges, with less prominent fluctuations of PHE levels over time.

“For the first time, the application of a pharmaceutical technology to a Medical Food brings a real change in the spectrum of the amino acid mixes, currently the main tool able to support PKU patients in their life-long necessity of controlling blood PHE levels. As a result, our solution, which aims at allowing patients to absorb a nutritionally adequate mix of amino acids in a more physiological manner, has the potential to modulate those unwanted and sometimes hidden disabilities expressed by some PKU patients, with plausible benefits on the quality of their lives.” Giorgio Reiner, APR Corporate Director R&D, states.

2017-04-27T15:18:45+00:00 Applied Pharma Research (APR)