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.
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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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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This post was originally published on Financial Sector