Pompe Disease Market Research Report by Leading Key Players –Genzyme Corporation, EpiVax,Amicus Therapeutics, Audentes Therapeutics, BioMarin, Valerion Therapeutics, Oxyrane - Pompe Disease Market Research Report by Leading Key Players –Genzyme Corpor

The pompe disease market is growing at a significant rate mainly due to special provisions for orphan drugs. Regulatory assistance in emerging nations is one of the major trends seen in the pompe disease market.

Pompe disease is a type of lysergic acid diethylamide (LSD). LSD refers to a group of diseases arising from the increase of specific substrates and is inherited autosomal recessive disorders. Lysergic acid diethylamide is progressive and having uneven age of onset and clinical symptoms. Pompe disease is a hereditary and repeatedly incurable disorder caused by lack of acid alpha glucosidase, an enzyme desired to split down glycogen in particular structures of our body.

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Pompe disease is categorized into two broad categories as adult-onset pompe disease and infantile onset pompe disease. Both the parents pass on one strangely altered copy of the gene to their child. A parent with an altered copy of the gene is known as a carrier and the disease can affect both men and women irrespective of the gender. The disease is acquired by the individuals at the time of birth only, but the onset of indications differs significantly. The researchers have identified more than 300 genetic mutations that can cause pompe disease. Enzyme replacement therapy and diet therapy are useful in dropping the clinical demonstrations of the disease.

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The competition in the pompe disease market is mainly weak, and the market is served by the two estrogen replacement therapy drugs available in the market namely Myozyme for infants and Lumizyme for adults. Both the drugs are permitted for different subclasses of Pompe disease. Gene therapy for the treatment of pompe disease is presently under study; however, its advancement is in a preclinical stage. Dietary treatment for pompe disease has also been described and it is under study.

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Government regulations, such as orphan drug exclusivity and limited patient population are the factors restraining the growth of the pompe disease market.

Some of the competitors in the market are Genzyme Corporation, EpiVax, Inc., Amicus Therapeutics, Audentes Therapeutics, BioMarin Pharmaceutical Inc., Valerion Therapeutics, LLC, Oxyrane, and Sangamo BioSciences Inc.

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