Portland, OR -- (SBWIRE) -- 11/09/2017 -- Pompe disease is a disorder caused by the buildup of a complex sugar called glycogen in the body's cells. It is genetically inherited and the accumulation of glycogen in certain organs as well as tissues, especially muscles, impairs their ability to function normally. Pompe diseases affects approximately one individual in 40,000 in the U.S.; however, the incidence varies among different ethnic groups.

Increasing research activities preformed to find an effective treatment for the disease and the growth in the special regulatory drug designations for orphan drugs drive the market. However, inflated costs associated with the Pompe disease therapy restrain the market growth. Moreover, emerging immunotherapeutic approaches for Pompe disease offer lucrative opportunities for the expansion of the market.

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The market is segmented on the basis of therapy type, molecule type, route of administration, dosage forms, and region. Based on therapy type, the market is divided into enzyme replacement therapy and substrate reduction therapy. Based on the molecule type, the market is classified into biologics and small molecules. Based on route of administration, the market is bifurcated into oral and parenteral.

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Key players that operate in the market are Amicus Therapeutics, BioMarin Pharmaceutical, Genzyme, Audentes Therapeutics, EpiVax, Oxyrane, Sangamo BioSciences, and Valerion Therapeutics.

Pompe Disease Market Key Segments:

By Therapy Type

-Enzyme Replacement Therapy
-Substrate Reduction Therapy

By Molecule Type

-Biologics
-Small Molecules

By Route of Administration

-Oral
-Parenteral

By Dosage Forms

-Solid
-Liquid