30 July 2020
Visiongain has launched a new report Pompe Disease Market Report 2020-2030: Forecasts by Therapy Type (Enzyme Replacement Therapy and Substrate Reduction Therapy), Dosage Forms (Solid and Liquid), End-user (Hospitals and Clinics, Research and Academic Institutes), and by Region (North America, Europe, Asia Pacific, Latin America & MEA), Including Forecasts by Major Developed Countries and Developing Countries, Plus Profiles of Leading Companies.
Pompe disease is a rare disease that affects approximately 1 in 40,000 births in the US. It is an autosomal recessively inherited metabolic disorder that affects one of the lysosomal enzymes leading to the deficiency of acid α-glucosidase. It is a fatal mascular disease that causes abnormal functioning of organs and tissues owing to the accumulation of glycogen in them. The mutation of gene that produces enzyme acid alpha-glycosidase prevents the breakage of lysosomal glycogen, leading to massive cellular dysfunction. This disease mainly affects the skeletal and cardiac muscles, resulting in skeletal muscles atrophy, respiratory distress, and cardiomyopathy.
Pompe disease can be diagnosed in various age groups and population across the globe. An infant affected by this disease has the survival rate of almost 26%. Some of the symptoms observed in infants affected by Pompe disease are heart defect, enlarged liver, large protruding tongue, muscle weakness, and poor muscle tone. The disease leads to weight gain in infants and causes problems related to growth and breathing. If left untreated, the infant may die in the first year due to heart failure. In some cases, the symptoms of Pompe disease such as poor motor skills and delay in muscle progress can be observed after the age of one. In this case, the patient suffers from hearing impairment, muscle weakness, and abnormally large heart. In cases where the patient shows symptoms in their adolescent age or adulthood, the symptoms are milder and does not led to any heart condition. However, the muscle weakness can cause breathing problems and lead to respiratory failure.
The advancement in gene therapy to treat Pompe disease, requirement of continuous uptake of medication, and launch of novel drugs boost the growth of global Pompe disease market. However, the high cost of these drugs is expected to hamper the market growth.
The Visiongain report analyst commented “The severity and fatal nature of Pompe disease and limited number of treatment options have led to an increase in the need to develop more effective drugs. The development of gene therapy and requirement for long-term treatment drives the growth of the Pompe Disease market”.
The report covers a detailed competitive outlook that includes market share and company profiles of key players operating in the global market. Key players profiled in the report include Genzyme, BioMarin Pharmaceutical, Amicus Therapeutics, Avrobio, Inc., Centogene AG, Audentes Therapeutics, EpiVax, Oxyrane, Sangamo BioSciences, and Valerion Therapeutics.
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