Propionic Acidemia (PA) – Market Outlook, Epidemiology, Competitive Landscape, and Market Forecast Report – 2023 To 2033
Propionic
acidemia (PA) is a rare organic aciduria caused by a propionyl Coenzyme A
carboxylase deficiency. This condition is characterized by severe metabolic
decompensation, neurological dysfunction, and potential complications such as
cardiomyopathy. PA can manifest in three distinct forms: severe neonatal onset,
intermittent late onset, or a chronic progressive form. In the severe neonatal
onset variant, affected infants display symptoms like poor feeding, vomiting,
and altered sensorium within hours to weeks after birth. PA results from
mutations in the PCCA and PCCB genes, which encode the propionyl CoA
carboxylase subunits. Newborn screening tests identify PA through elevated
propionyl carnitine levels, and differential diagnosis involves distinguishing
it from other conditions like neonatal sepsis and other organic acidurias. In
the infantile chronic form, symptoms can mimic those of cow milk intolerance,
celiac disease, or immune deficiencies, including failure to thrive, chronic
vomiting, and neutropenia. Early detection and treatment have improved survival
rates in early and mid-childhood, although cognitive development impairment
remains common. Hyperammonemia, a critical complication, can be managed with
sodium benzoate, carbamyl glutamate, or hemodialysis. Long-term treatment
primarily involves nutritional management, including protein restriction.
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Propionic acidemia (PA) affects roughly 1 in
100,000 people in the United States.
Thelansis’s
“Propionic Acidemia (PA) 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 Propionic
Acidemia (PA) treatment modalities options for eight major markets (USA,
Germany, France, Italy, Spain, UK, Japan, and China).
KOLs insights of Propionic Acidemia
(PA) 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.
Propionic
Acidemia (PA) 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: Propionic
Acidemia (PA), Propionic Acidemia (PA) market
outlook, Propionic Acidemia (PA) competitive
landscape, Propionic Acidemia (PA) market forecast,
Thelansis, Primary market research, KOL insights, Competitive Intelligence (CI)
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