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Treatments Meant for Pompe Disease
Pompe disease is a progressive inherited disease which is debilitating, multisystemic, and often fatal neuromuscular disorder.This most rare disease was first identified in the year 1932 by Joannes C. Pompe, a Dutch pathologist in a seven-month old infant who died of idiopathic cardiac hypertrophy and after examining it was found that the child has excessive accumulation of glycogen in several tissues, however predominant in cardiac and skeletal muscles. Later in 1963, Pompe disease was associated with a genetic deficiency of the lysosomal enzyme acid alpha-glucosidase (GAA), which is accountable for the glycogen to breaking down to glucose.This process leads to intralysosomal accumulation of glycogen, mainly in muscle cells, which eventually results in gradual loss of muscle function. Study done on Pompe disease treatment market states that researchers have characterized three types of Pompe disease, which alter in rigorousness and the individual’s age at which they surface. They are classified into infantile-onset, non-classic infantile-onset, and late-onset.There are many synonyms for Pompe disease such as acid maltase deficiency (AMD), acid alpha glucosidase (GAA) deficiency, and glycogen storage disease type II.
When does Pompe disease happen?
This disease occurs when the human body cannot generate protein which further breaks down a complex sugar, known as glycogen, meant to give energy. As a result, excessive accumulation of sugar occurs which hampers and damages the organs and muscles. Pompe disease makes the muscles extremely weak and the patient might have trouble breathing. It has been observed that this disease affects the heart, liver and muscles. Even though it can affect anyone, it is more prevalent in African-American individuals and also a few Asian groups. A person gets this disease from his or her parents. In order to get it, an individual must have inherited two flawed genes, one from each parent. However, a person can just have one gene and not possess any symptoms of the disease.
Pompe Disease Treatment
The treatment meant for Pompe disease is an amalgamation of both supportive therapies and disease-specific. Generally, the treatment might need the combined efforts of experts who specialize in treating neuromuscular disorders.For a child having this disease, pediatricians, orthopedists, neurologists, dieticians, cardiologists and many other healthcare experts might have to be consistently and all inclusive strategize a child’s treatment. The United States Food and Drug Administration (FDA) in 2006, approved the enzyme replacement therapy Myozyme for patients who were suffering from infantile onset Pompe disease. Later in 2010, Lumizyme was approved. Usually for patients who are younger than eight years of age, are given Myozyme; and for older patients it is Lumizyme. Healthcare professionals suggest enzyme replacement therapy every two weeks by administering intravenously of genetically engineered human acid alpha-glucosidase at a dosage of 20mg/kg of the patient’s body weight.Traditionally, treatments meant for this disease has aimed more on managing the many symptoms and rendering utmost supportive care, and these methods continue to be crucial elements of the complete approach of treatment.
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Table of Contents
United States Pompe Disease Treatment Market Report 2017
1 Pompe Disease Treatment Overview
1.1 Product Overview and Scope of Pompe Disease Treatment
1.2 Classification of Pompe Disease Treatment
1.2.1 Infantile-Onset Pompe Disease
1.2.2 Adult-Onset Pompe Disease
1.3 Application of Pompe Disease Treatment
1.4 United States Market Size Sales (Volume) and Revenue (Value) of Pompe Disease Treatment (2012-2022)
1.4.1 United States Pompe Disease Treatment Sales and Growth Rate (2012-2022)
1.4.2 United States Pompe Disease Treatment Revenue and Growth Rate (2012-2022)
2 United States Pompe Disease Treatment Competition by Manufacturers
2.1 United States Pompe Disease Treatment Sales and Market Share of Key Manufacturers (2015 and 2016)
2.2 United States Pompe Disease Treatment Revenue and Share by Manufactures (2015 and 2016)
2.3 United States Pompe Disease Treatment Average Price by Manufactures (2015 and 2016)
2.4 Pompe Disease Treatment Market Competitive Situation and Trends
2.4.1 Pompe Disease Treatment Market Concentration Rate
2.4.2 Pompe Disease Treatment Market Share of Top 3 and Top 5 Manufacturers
2.4.3 Mergers & Acquisitions, Expansion
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