A Neurometabolic disorder is a group of individually rare and progressive disorders limited to symptomatic patient care. The analytical and clinical research result predicts significant development in explaining the molecular genetics, biochemical irregularities, pathogenesis of this disorder and diagnostic testing. The major symptoms of neurometabolic disorders are caused by progressive destruction of mental, motor and perceptual functions. These disorders also include seizures and loss of function associated with early death (before adulthood). Neurometabolic disorders primarily affect multiple organs and their symptoms vary from acute life-threatening disease to sub-acute progressive degenerative disorders.
The most common neurometabolic disorders are amino acid apathies and organic acidurias followed by lipofuscinoses, congenital lactic acidosis, neuronal ceroid urea cycle disorders, peroxisomal disorders, while the less frequent are glycoprotein, mucopolysaccharidoses, degradation disorders, sphingolipidoses, and fatty acid oxidation disorders. The neurometabolic disorders market can be segmented on the basis of various disorders such as Pompe Disease, Fabry Disease, Gaucher’s Disease, Niemann-Pick Type C Disease and Mucopolysaccharidosis VI.
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Gaucher’s disease is a genetic disorder in which lipid (fatty substance) accumulates in the cells and certain organs. The disorder is differentiated by fatigue, low blood platelets, anemia, bruising, enlargement of liver and spleen. The major products used in treatment of Gaucher’s disease are Cerezyme manufactured by Genzyme (subsidiary of Sanofi SA) and VPRIV (velaglucerase alfa for injection) manufactured by Shire Plc. Pompe Disease is one of the rarest diseases, hereditary and often lethal disorder that disables the skeletal and heart muscles. It causes mutation in acid alpha-glucosidase (GAA) enzyme producing gene that tends to accumulate excess amounts of lysosomal glycogen mount up in various parts of the body, cardiac and skeletal muscles are the most fatally affected. The products used in treatment of Pompe Disease are Myozyme/Lumizyme manufactured by Genzyme (subsidiary of Sanofi SA). Fabry disease is a rare form of genetic lysosomal storage disease leading to deficiency in enzyme alpha galactosidase A (a-GAL A) that accounts for the associated clinical abnormalities of brain, eye, skin, heart, kidney and peripheral nervous system. The treatment regime of Fabry disease includes Fabrazyme that is manufactured by Genzyme (subsidiary of Sanofi SA) and Replagal manufactured by Shire Plc. Niemann-Pick Type C Disease is a lysosomal storage disease related with the mutations in NPC-1 and NPC-2 genes where the transport system related with cholesterol and glycolipids is disrupted and which leads to their excessive accumulation.
The treatment for Niemann-Pick Type C includes Zavesca a product of Actelion Pharmaceuticals. Mucopolysaccharidosis VI consists of malfunctioned activity of the lysosomal enzymes, which interrupts the degradation of mucopolysaccharides which leads to abnormal buildup of keratan sulfate, dermatan sulfate, heparan sulfate. The standard treatment against mucopolysaccharidosis VI is with Naglazyme a product of BioMarin Pharmaceutical, Inc.
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The growth in institutional and research fields form transcriptomics, proteomics, metabolomics and lipidomics is expected to develop more advanced diagnostics and treatment. The various economic leverages provided by USFDA for orphan drug and diagnostics manufacturing companies are expected to grow the market. Various institutional bodies investing the knowledge and economic sources in orphan disease research incur funding from various sources that is expected to contribute to sort out the role of very rarely studied organelle in neurodegenerative diseases and aging.
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Some of the major players in the Neurometabolic disorders market are Shire Plc, BioMarin Pharmaceutical, Inc., Actelion Pharmaceuticals Ltd., Sanofi SA, Pfizer, Inc., GlaxoSmithKline, Amicus Therapeutics, Inc. and others. The neurometabolic disorders market was dominated by Sanofi SA, GlaxoSmithKline, Amicu Therapeutics, Inc, and Pfizer, Inc.
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