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.
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.
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.
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.
This research report analyzes this market on the basis of its market segments, major geographies, and current market trends. Geographies analyzed under this research report include
- North America
- Asia Pacific
- Rest of the World
This report provides comprehensive analysis of
- Market growth drivers
- Factors limiting market growth
- Current market trends
- Market structure
- Market projections for upcoming years
This report is a complete study of current trends in the market, industry growth drivers, and restraints. It provides market projections for the coming years. It includes analysis of recent developments in technology, Porter’s five force model analysis and detailed profiles of top industry players. The report also includes a review of micro and macro factors essential for the existing market players and new entrants along with detailed value chain analysis.
Reasons for Buying this Report
- This report provides pin-point analysis for changing competitive dynamics
- It provides a forward looking perspective on different factors driving or restraining market growth
- It provides a six-year forecast assessed on the basis of how the market is predicted to grow
- It helps in understanding the key product segments and their future
- It provides pin point analysis of changing competition dynamics and keeps you ahead of competitors
- It helps in making informed business decisions by having complete insights of market and by making in-depth analysis of market segments
- It provides distinctive graphics and exemplified SWOT analysis of major market segments
Note : All statements of fact, opinion, or analysis expressed in reports are those of the respective analysts. They do not necessarily reflect formal positions or views of Transparency Market Research.