Barth Syndrome – Market Outlook, Epidemiology, Competitive Landscape, and Market Forecast Report – 2023 To 2033 thelansis.com

Barth syndrome (BTHS) is a rare X-linked genetic disorder that has distinct characteristics, including cardiomyopathy (CM), skeletal myopathy, growth delay, neutropenia, and elevated urinary levels of 3-methylglutaconic acid (3-MGCA). This condition arises from mutations in the TAZ gene (at Xq28), which encodes Taz1p acyltransferase, an enzyme crucial for cardiolipin metabolism—a major phospholipid in the inner mitochondrial membranes. Dysfunctional Taz1p leads to abnormal cardiolipin remodeling, compromising the integrity of mitochondrial structure and respiratory chain function. Clinical manifestations encompass a broad spectrum, with varying combinations of the following features: dilated cardiomyopathy (DCM), left ventricular non-compaction (LVNC), endocardial fibroelastosis (EFE), hypertrophic cardiomyopathy (HCM), ventricular arrhythmia, sudden cardiac death, neutropenia (ranging from absent to severe), delayed motor milestones, proximal myopathy, lethargy, fatigue, prolonged QTc interval, failure to thrive, compensatory monocytosis, recurrent bacterial infections, hypoglycemia, feeding difficulties, growth and pubertal delays, lactic acidosis, intermittent or perfectly cyclical symptoms like diarrhea, characteristic facial features, and a family history of X-linked inheritance. The average age at which BTHS is diagnosed falls between 4 and 5 years, even though symptoms typically occur between 0.7 and 1.5 years. Cardiomyopathy accounts for 70% of the primary reasons for pursuing DNA diagnostic testing to confirm BTHS. Other reported motivations for testing include neutropenia, growth delay/failure to thrive, and a family history of the condition.
• In the United States, around 10 new cases are diagnosed annually, and 71 distinct family lines have been identified. The Barth Syndrome Foundation (BSF) estimates that the prevalence of BTHS is approximately 1 in 300,000 to 400,000 live births in the USA.

Thelansis’s “Barth Syndrome 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 Barth Syndrome treatment modalities options for eight major markets (USA, Germany, France, Italy, Spain, UK, Japan, and China).

KOLs insights of Barth Syndrome 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.
Barth Syndrome 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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