Phenylketonuria (PKU) – Market Outlook, Epidemiology, Competitive Landscape, and Market Forecast Report – 2021 To 2032


 

Phenylketonuria (PKU) is an autosomal recessive inborn error of phenylalanine (Phe) metabolism resulting from a deficiency of phenylalanine hydroxylase (PAH). Most forms of PKU and hyperphenylalaninaemia (HPA) are caused by mutations in the PAH gene on chromosome 12q23.2. Untreated PKU is associated with an abnormal phenotype, including seizures, poor skin pigmentation, microcephaly, growth failure, global developmental delay and severe intellectual impairment. However, since the introduction of newborn screening programs and early dietary intervention, children born with PKU can now expect to lead relatively normal lives. Pharmacological treatment with BH4 (sapropterin) and enzyme substitution therapy with Phe ammonia lyase (PAL) provide alternative treatment options for PKU patients.

  • In the United States, the reported incidence of PKU varies between 1 to 2 cases in every 20,000 newborns.

Thelansis’s “Phenylketonuria (PKU) Market Outlook, Epidemiology, Competitive Landscape, and Market Forecast Report – 2021 To 2032" covers disease overview, epidemiology, drug utilization, prescription share analysis, competitive landscape, clinical practice, regulatory landscape, patient share, market uptake, market forecast, and key market insights under the potential Phenylketonuria (PKU) treatment modalities options for eight major markets (USA, Germany, France, Italy, Spain, UK, Japan, and China).

KOLs insights of Phenylketonuria (PKU) across 8 MM market from the centre of Excellence/ Public/ Private hospitals participated in the study. Insights around current treatment landscape, epidemiology, clinical characteristics, future treatment paradigm, and Unmet needs.

Phenylketonuria (PKU) Market Forecast Patient Based Forecast Model (MS. Excel Based Automated Dashboard), which Data Inputs with sourcing, Market Event, and Product Event, Country specific Forecast Model, Market uptake and patient share uptake, Attribute Analysis, Analog Analysis, Disease burden, and pricing scenario, Summary, and Insights.

Thelansis Competitive Intelligence (CI) practice has been established based on a deep understanding of the pharma/biotech business environment to provide an optimized support system to all levels of the decision-making process. It enables business leaders in forward-thinking and proactive decision-making. Thelansis supports scientific and commercial teams in seamless CI support by creating an AI/ ML-based technology-driven platform that manages the data flow from primary and secondary sources.

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