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Results for tag: "Barth Syndrome"

Leber hereditary optic neuropathy - Pipeline Insight, 2024 - Product Thumbnail Image

Leber hereditary optic neuropathy - Pipeline Insight, 2024

  • Drug Pipelines
  • April 2024
  • 60 Pages
  • Global
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Barth Syndrome - Pipeline Insight, 2021 - Product Thumbnail Image

Barth Syndrome - Pipeline Insight, 2021

  • Drug Pipelines
  • September 2021
  • 60 Pages
  • Global
From
Barth Syndrome - Pipeline Review, H2 2018 - Product Thumbnail Image

Barth Syndrome - Pipeline Review, H2 2018

  • Drug Pipelines
  • July 2018
  • 42 Pages
  • Global
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  • 5 Results (Page 1 of 1)
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Barth Syndrome is a rare genetic disorder that affects the cardiovascular system. It is caused by a mutation in the TAZ gene, which is responsible for the production of a protein called tafazzin. Symptoms of Barth Syndrome include cardiomyopathy, skeletal myopathy, neutropenia, and growth retardation. Treatment for Barth Syndrome is focused on managing the symptoms, and may include medications to reduce inflammation, improve cardiac function, and reduce the risk of infection. In addition, lifestyle modifications such as diet and exercise may be recommended. The Barth Syndrome market is a small but growing segment of the Cardiovascular Drugs market. As the understanding of the disorder increases, more treatments are being developed to address the symptoms. Companies in the Barth Syndrome market include Akcea Therapeutics, Apellis Pharmaceuticals, and Ultragenyx Pharmaceuticals. Show Less Read more