Barth Syndrome – Market Outlook, Epidemiology, Competitive Landscape, and Market Forecast Report – 2021 To 2032

 

Barth syndrome (BTHS) is widely recognised as a rare X-linked genetic disease characterised by cardiomyopathy (CM), skeletal myopathy, growth delay, neutropenia, and increased urinary excretion of 3- methylglutaconic acid (3-MGCA). Clinical features include variable combinations of the following wide spectrum: dilated cardiomyopathy (DCM), left ventricular non-compaction (LVNC), endocardial fibroelastosis (EFE), hypertrophic cardiomyopathy (HCM), ventricular arrhythmia, sudden cardiac death, neutropenia (absent to severe; persistent, delayed motor milestones, proximal myopathy, lethargy and fatigue, prolonged QTc interval, failure to thrive, compensatory monocytosis, recurrent bacterial infection, hypoglycaemia, feeding problems, growth and pubertal delay, lactic acidosis, intermittent or perfectly cyclical), episodic diarrhoea, characteristic facies, and X-linked family history.

• The mean age at diagnosis of BTHS was 4 to 5 years, although the age of onset of symptoms was 0.7 to 1.5 years. The primary reason for further DNA diagnostic testing confirming BTHS was CM (70%), with other reported reasons including neutropenia, growth delay/failure to thrive and family history.
• The majority (64%) had missense substitutions, 16% had partial gene deletions, 12% had partial gene duplications, and fewer than 5% had a complete gene deletion, nonsense mutation, or splice site mutation.

Thelansis’s “Barth Syndrome 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 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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