Pediatric Clinics Phenylketonuria Market is a rare genetic disorder that affects the way the body processes the amino acid phenylalanine. PKU is caused by a deficiency in the enzyme phenylalanine hydroxylase (PAH), which is responsible for converting phenylalanine to tyrosine. When a person with PKU consumes foods containing phenylalanine, the amino acid builds up in their blood and can lead to serious health problems, including intellectual disability, seizures, and behavioral issues.

The PKU market is relatively small, with an estimated prevalence of 1 in 10,000 to 1 in 15,000 births worldwide. The current standard of care for PKU involves a strict low-phenylalanine diet, which can be challenging to adhere to and may not provide sufficient levels of other essential nutrients. In recent years, several pharmaceutical companies have developed treatments for PKU, including enzyme replacement therapies and gene therapies.

One of the most promising treatments for PKU is a drug called pegvaliase (Palynziq), which was approved by the US Food and Drug Administration (FDA) in 2018. Pegvaliase is an enzyme replacement therapy that works by breaking down phenylalanine in the body. The drug is administered through subcutaneous injections and has been shown to significantly reduce blood phenylalanine levels in patients with PKU. Another promising therapy for PKU is gene therapy, which aims to restore the function of the PAH enzyme in patients with the disorder. In 2019, a team of researchers from the University of Pennsylvania reported successful results from a phase 1/2 clinical trial of a gene therapy for PKU. The therapy involves using a harmless virus to deliver a functional copy of the PAH gene to liver cells. The researchers reported that the therapy was well-tolerated and led to a significant reduction in blood phenylalanine levels in the study participants.

Request a sample Report of Phenylketonuria (PKU) Market https://www.reportsanddata.com/download-free-sample/319

In addition to these emerging therapies, several companies are working on developing new treatments for PKU. BioMarin Pharmaceutical is currently developing a drug called vosoritide, which is designed to treat achondroplasia, a rare genetic disorder that causes short stature. The company is also exploring the potential of vosoritide as a treatment for PKU, based on its ability to modulate the growth hormone pathway, which is thought to be involved in the regulation of phenylalanine metabolism.

Other companies, such as Homology Medicines and Ultragenyx Pharmaceutical, are also developing gene therapies for PKU. Homology Medicines is developing a gene therapy that involves using a proprietary technology called meganuclease to edit the PAH gene in liver cells. Ultragenyx Pharmaceutical is developing a gene therapy that uses a harmless virus to deliver a functional copy of the PAH gene to liver cells.In conclusion, while the PKU market is relatively small, there is significant interest and investment in developing new treatments for this rare genetic disorder. Enzyme replacement therapies and gene therapies show promise in reducing blood phenylalanine levels in patients with PKU and improving outcomes. As research in this area continues to advance, there is hope that new treatments will become available to help improve the lives of people with PKU.

The key driving factors of the Phenylketonuria (PKU) market include:

• Increased awareness: There has been a rise in awareness amongst people regarding Phenylketonuria globally. This has led to an increase in demand for PKU treatment and is driving the growth of the market.

• Advancements in healthcare industry: The healthcare industry has been witnessing significant advancements in the treatment of PKU. These advancements are helping to improve the quality of life for PKU patients and are also driving the growth of the market.

• Rising frequency of PKU in infants: The rising frequency of PKU in infants is driving the demand for PKU treatment. This is expected to continue to drive the growth of the market over the forecast period.

• Increased research in the field of genomics and bioinformatics: Increased research in the field of genomics and bioinformatics is leading to the development of new drugs for the treatment of PKU. This is driving the growth of the market.

• PKU awareness programs: PKU awareness programs sponsored by local governments, NGOs, and regulatory agencies, as well as healthcare system help in established and emerging nations, are driving the growth of the market.

Request a customized copy of the report https://www.reportsanddata.com/request-customization-form/319

About Reports and Data 

Reports and Data is a market research and consulting company that provides syndicated research reports, customized research reports, and consulting services. Our solutions purely focus on your purpose to locate, target, and analyze consumer behavior shifts across demographics, across industries, and help clients to make smarter business decisions. We offer market intelligence studies ensuring relevant and fact-based research across multiple industries, including Healthcare, Touch Points, Chemicals, Products, and Energy. We consistently update our research offerings to ensure our clients are aware of the latest trends existent in the market. Reports and Data has a strong base of experienced analysts from varied areas of expertise. Our industry experience and ability to develop a concrete solution to any research problems provides our clients with the ability to secure an edge over their respective competitors.

Contact:

John W, Head of Business Development
Reports And Data | Web: www.reportsanddata.com
Direct Line: +1-212-710-1370
E-mail: sales@reportsanddata.com
LinkedIn | Twitter | Blogs