Phenylketonuria (PKU) is an inborn error of amino acid phenylalanine metabolism caused mainly by PAH gene mutations. Dietary interventions were considered to be a good approach to phenylketonuria treatment in preventing various clinical features, especially the intellectual disability in patient’s populations. However low rate of adherence to dietary therapy has notably led to suboptimal outcomes. Another way of fulfilling the metabolic deficiencies is through genetically modified (GM) probiotics that can help delivery of metabolic enzymes.
Substantial advances in gene therapy have invigorated the growth in the phenylketonuria treatment market over the years along with the advent of enzyme-based therapies. Industry search for new treatment strategies also has been focusing on bolstering the efficacy of oral therapy, mainly by phenylanine ammonia-lyase. Efforts have also been made to increase the palatability of medical foods.
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Recent developments in PKU treatments have been stirred increasingly by the morbidity of various clinical features that are associated with untreated individuals. Most prominent clinical manifestations that surface with age in patient populations are behavioral impairment and psychiatric disturbances.
Gene Therapy and Enzyme-based Therapies emerging as Potentially Promising Alternatives to Dietary Interventions
Growing number of newborn screening programs in various developing and developed countries of Europe and North America has helped boost the phenylketonuria treatment market prospects. In recent years, several alternative treatments have emerged in the phenylketonuria treatment market, more notably gene therapy associated with modification in adenoviral vectors. Of the various therapies, perhaps the one showing the highest potential is novel enzyme replacement therapy. Infusion of PAH-based fusion proteins has shown good potential in clinical trials on mice. However, the clinical translation of such therapy will undoubtedly need more research involving models orthologous to humans.
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Another approach that might play crucial role in changing the course of the phenylketonuria treatment market is enzyme substitution therapy. Advances in therapeutic approaches have laid great emphasis on this therapy. This is accomplished by phenylalanine ammonia-lyase enzyme. Growing number of studies in the past three decades has helped stimulate industry developments in this direction.