Pompe disease is a genetic metabolic disorder that occurs in infants. This disease is caused due to the mutations in the GAA gene responsible for producing the acid, alpha-glucosidase enzyme that converts glycogen into a simple form. The absence or mutation in the GAA gene leads to the accumulation of glycogen and results to have a heart problem, muscle weakness, liver damage that can lead to premature death in the new-born.

Global Pompe Disease Treatment Market Share is expected to register a CAGR of 3.0% during the forecast period and expected to hit USD 1,664.9 Million by 2030.

The increasing government initiatives and rising R&D activities by manufacturers to find new solutions for the diagnosis and treatment of Pompe disease are anticipated to drive the global Pompe disease treatment market growth. However, the high cost of treatment is expected to restrict market growth to a certain extent.

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Key Players

MRFR recognizes the following companies as the Key Players in the Global Pompe Disease Treatment Market— Amicus Therapeutics, Inc (US) Sanofi (France), Audentes Therapeutics (US), Oxyrane (UK), Valerion Therapeutics (US), AVROBIO, Inc. (US), CENTOGENE AG (UK), and others.

Segment Analysis

The global Pompe disease treatment market has been divided based on type, therapy, and end-user.

Based on Type has been segregated into late-onset Pompe disease, classic infantile-onset Pompe disease, and non-classic infantile-onset Pompe disease.
Based on Therapy, the Pompe disease treatment market has been divided into enzyme replacement therapy (ERT), gene therapy, and others.
Based on End-user, the market is bifurcated into hospitals & clinics and research & academic institutes.
Regional Analysis

The global Pompe disease treatment market, based on region, has been divided into the Americas, Europe, Asia-Pacific, and the Middle East & Africa. The Americas is likely to hold the largest share of the global Pompe disease treatment market during the assessment period. This largest share can be attributed to the presence of established payers and an increase in the number of patients with Pompe disease in the region. The American region is a hub for major market players such as Genzyme Corporation, Audentes Therapeutics, and Amicus Therapeutics, Inc operating in the Pompe disease treatment market, which eventually drives the growth of the regional market. Additionally, the growing awareness among the general population regarding the available treatments for Pompe disease is projected to boost the regional market growth during the review period.

Europe is the second-largest market for Pompe disease treatment owing to factors such as the growing research and development initiatives, support by the governments in the healthcare sector, and improvement in reimbursement policies. For instance, European countries are extremely cautious and have implemented the newborn screening (NBS) program as a means of early detection and identification of severe illnesses in newborns, including Pompe disease.

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Asia-Pacific is estimated to be the fastest-growing region in the global market due to the increasing healthcare expenditure, growing awareness among patients regarding rare disorders, and expanding healthcare industry. The Pompe disease treatment market in the Middle East & Africa is expected to witness gradual growth due to the significantly grow owing to the growing awareness about the treatment of rare diseases and developing healthcare infrastructure.