New Treatments Emerge for Phenylketonuria Phenylketonuria (PKU) is an inherited metabolic disorder characterized by the body's inability to properly process the essential amino acid phenylalanine. Without treatment, high phenylalanine levels can cause intellectual disabilities and other neurological problems. PKU affects approximately 1 in 15,000 newborns in the United States each year.
Diagnosis and Current Treatment Options
PKU is diagnosed through newborn screening that measures phenylalanine levels in a blood sample taken shortly after birth. If elevated levels are found, further testing is done to confirm the diagnosis. The standard Phenylketonuria Treatment involves following a lifelong low-phenylalanine diet. This requires avoiding foods containing phenylalanine, such as meat, poultry, fish, eggs, dairy products, and many baked goods. Synthetic formula and nutritional supplements help provide adequate nutrition while keeping phenylalanine intake very low. Strict diet adherence is necessary to prevent cognitive and developmental issues.
Alternative Treatment: Kuvan
For patients who have difficulty maintaining the restrictive diet, an alternative treatment option called sapropterin dihydrochloride (Kuvan) was approved in 2007. Kuvan works by increasing the body's ability to process phenylalanine. Clinical trials showed it effectively reduced phenylalanine levels in some patients when taken in conjunction with a phenylalanine-restricted diet. For those who respond well, Kuvan allows for a less stringent diet and improved dietary flexibility. However, not all PKU patients see the same benefits from Kuvan treatment. More research continues to better understand its long-term effects and which patient groups are most likely to respond.
New Medications on the Horizon
While Kuvan was a milestone, researchers recognize the need for additional treatment options. Several companies are exploring new therapies with the goal of normalizing phenylalanine control without requiring strict, lifelong dietary management. Some potential new treatments in development include:
Phenylacetate-MonoGlycine Conjugate: Developed by Synlogic, this investigational oral therapy is designed to help the body break down and eliminate excess phenylalanine in a similar way to Kuvan. Phase 2 trials are currently underway.
PN-935: This oral medication from Premier Research is aimed at blocking phenylalanine uptake in the intestine. Phase 1 trials showed it effectively reduced phenylalanine levels. Phase 2 trials will evaluate its potential as a diet relaxation agent.
Gene Therapy: Researchers are also investigating gene therapy as a cure for PKU. One approach involves using an engineered virus to deliver a working copy of the PAH gene directly into liver cells. Early animal studies showed the modified gene was successfully expressed long-term. Human trials are still years away but offer hope as a onetime treatment.
Diet Compliance Challenges Persist
While new medications continue advancing, diet remains the backbone of PKU treatment. But getting patients, especially adolescents and young adults, to consistently stick to the restrictive regimen poses ongoing challenges. Forgetfulness, lack of variety, social pressures, and the desire for independence all contribute to nonadherence over time. Diet relapses put patients at risk for neurological problems later in life now that treatment options exist to delay symptoms. Healthcare providers emphasize the importance of transition planning and ongoing support as patients mature to help them successfully integrate PKU management into independent living. Transition clinics catering specifically to this age group aim to smooth the handoff of responsibility. With emerging therapies and a focus on transition support, PKU patients now have more options than ever before to optimize treatment outcomes long-term.
the understanding and treatment of PKU has come a long way since its discovery as an inherited disorder. While existing therapies like Kuvan provide more flexibility, emerging new medications hold promise as alternatives or additions to the low-phenylalanine diet. With a variety of treatment approaches in development and a focus on improving adherence and transitions of care, patients now have greater tools and support available to effectively manage PKU throughout life. Continued research remains vital to developing innovations that could one day lead to a cure and eliminate the lifelong challenges of this treatable condition.
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This report studies Phenylketonuria (PKU) in Global market, especially in North America, China, Europe, Southeast Asia, Japan and India, with production, revenue, consumption, import and export in these regions, from 2013 to 2018, and forecast to 2025This report focuses on top manufacturers in global market, with production, price, revenue and market share for each manufacturer, coveringRequest Free Sample Report: https://www.qymarketinsights.com/report-detail/37110/request-sampleBiomarin(US)Vitaflo(UK)Mead Johnson(US)Nutricia(US)Abbott(US)Prominmetabolics(US)Cambrooke(US)Juvela(UK)Firstplay Dietary(UK)On the basis of product, this report displays the production, revenue, price, market share and growth rate of each type, primarily split intoMedicationsSupplementsBy Application, the market can be split intoHouseholdHospitalBy Regions, this report covers (we can add the regions/countries as you want)North AmericaChinaEuropeSoutheast AsiaJapanIndiaAccess Full Report: https://www.qymarketinsights.com/report/global-phenylketonuria-pku-market-professional-survey-report-2018-37110.htmlIf you have any special requirements, please let us know and we will offer you the report as you want.Table of ContentsGlobal Phenylketonuria (PKU) Market Professional Survey Report 20181 Industry Overview of Phenylketonuria (PKU)1.1 Definition and Specifications of Phenylketonuria (PKU)1.1.1 Definition of Phenylketonuria (PKU)1.1.2 Specifications of Phenylketonuria (PKU)1.2 Classification of Phenylketonuria (PKU)1.2.1 Medications1.2.2 Supplements1.3 Applications of Phenylketonuria (PKU)1.3.1 Household1.3.2 Hospital1.3.3 Application 31.4 Market Segment by Regions1.4.1 North America1.4.2 China1.4.3 Europe1.4.4 Southeast Asia1.4.5 Japan1.4.6 India2 Manufacturing Cost Structure Analysis of Phenylketonuria (PKU)2.1 Raw Material and Suppliers2.2 Manufacturing Cost Structure Analysis of Phenylketonuria (PKU)2.3 Manufacturing Process Analysis of Phenylketonuria (PKU)2.4 Industry Chain Structure of Phenylketonuria (PKU)Contact UsMark StoneSales ManagerPhone: (201) 465-4211Email: sales@qymarketinsights.comWeb: https://www.qymarketinsights.com/
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