Primary Hyperoxaluria Type 1 (PH1) – Market Outlook, Epidemiology, Competitive Landscape, and Market Forecast Report – 2022 To 2032


 Primary hyperoxaluria type 1 (PH1) is a rare genetic disease caused by mutations in the AGXT gene, inherited in an autosomal recessive manner. This condition inflicts irreversible harm on the kidneys and other vital organs, carrying the potential for life-threatening consequences. PH1 results from a deficiency of the liver peroxisomal enzyme called alanine: glyoxylate-aminotransferase (AGT). AGT plays a crucial role in converting glyoxylate into glycine. Individuals affected by PH1 are susceptible to recurrent nephrolithiasis, which is the deposition of calcium oxalate in the renal pelvis and urinary tract, nephrocalcinosis, characterized by calcium oxalate buildup in the renal parenchyma, and end-stage renal disease (ESRD). Roughly 10% of those with PH1 experience symptoms during infancy or early childhood, including nephrocalcinosis, sometimes nephrolithiasis, and growth issues due to renal failure. Most individuals with PH1 are diagnosed during childhood or early adolescence, often presenting with symptomatic nephrolithiasis and either normal or diminished kidney function. PH1 diagnosis is confirmed in individuals showing hyperoxaluria or hyperoxaluria through genetic testing that identifies biallelic pathogenic variants in AGXT. The primary approach to preventing and treating the renal and systemic complications of PH1 is to reduce the liver's excessive oxalate production. Two main options are available: pharmacologic doses of pyridoxine (restricted to those whose AGXT genotype responds to pyridoxine) or liver transplantation, frequently combined with or sequentially performed alongside kidney transplantation in ESRD cases. In 2020, the FDA and EMA approved lumasiran, an mRNA therapeutic agent that reduces glyoxylate substrate levels available for metabolic conversion to oxalate.

Thelansis’s “Primary Hyperoxaluria Type 1 (PH1) Market Outlook, Epidemiology, Competitive Landscape, and Market Forecast Report – 2022 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 Primary Hyperoxaluria Type 1 (PH1) treatment modalities options for eight major markets (USA, Germany, France, Italy, Spain, UK, Japan, and China).

KOLs insights of Primary Hyperoxaluria Type 1 (PH1) 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.

Primary Hyperoxaluria Type 1 (PH1) 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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