Barth Syndrome – Market outlook, Epidemiology, Competitive Landscape and Market Forecast Report – 2020 To 2030
Barth syndrome is a metabolic and neuromuscular disorder, occurring almost exclusively in males, that primarily affects the heart, immune system, muscles, and growth. Etiology- Barth syndrome is caused by mutations in the TAZ gene, which is located on the X chromosome. The TAZ gene provides "instructions" for a group of proteins called tafazzins that serve at least two functions. Epidemiology- Barth syndrome is estimated to affect 1 in 300,000 to 400,000 individuals worldwide. The competitive landscape of Barth Syndrome includes country-specific approved as well as pipeline therapies. Any asset/product-specific designation or review and Accelerated Approval are being tracked and supplemented with analyst commentary. KOLs insights of Barth Syndrome across 8 MM market from the center of Excellence/ Public/ Private hospitals participated in the study. Insights around current treatment landscape, epidemiology, clinical characteristics, future t...