Phenylketonuria (PKU) – Market Outlook, Epidemiology, Competitive Landscape, and Market Forecast Report – 2023 To 2033
Phenylketonuria (PKU) is a genetic disorder characterized by a deficiency in phenylalanine hydroxylase (PAH), resulting in impaired phenylalanine (Phe) metabolism. Most cases of PKU and hyperphenylalaninaemia (HPA) stem from mutations in the PAH gene located on chromosome 12q23.2. If left untreated, PKU leads to an abnormal phenotype, including seizures, diminished skin pigmentation, microcephaly, growth retardation, global developmental delay, and severe intellectual impairment. However, thanks to the implementation of newborn screening programs and early dietary interventions, children born with PKU can now anticipate relatively normal lives. PKU patients also have alternative treatment options, such as pharmacological treatment with BH4 (sapropterin) and enzyme substitution therapy involving Phe ammonia lyase (PAL).
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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 – 2023 To 2033" 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.
Tags: Phenylketonuria
(PKU), Phenylketonuria (PKU) market outlook, Phenylketonuria
(PKU) competitive landscape, Phenylketonuria
(PKU) market forecast, Thelansis, Primary market
research, KOL insights, Competitive Intelligence (CI)
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