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 study by TMR is all-encompassing framework of the dynamics of the market. It mainly comprises critical assessment of consumers' or customers' journeys, current and emerging avenues, and strategic framework to enable CXOs take effective decisions.

Our key underpinning is the 4-Quadrant Framework EIRS that offers detailed visualization of four elements:

  • Customer Experience Maps
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  • Strategic Frameworks to boost the growth journey

The study strives to evaluate the current and future growth prospects, untapped avenues, factors shaping their revenue potential, and demand and consumption patterns in the global market by breaking it into region-wise assessment.

The following regional segments are covered comprehensively:

  • North America
  • Asia Pacific
  • Europe
  • Latin America
  • The Middle East and Africa

The EIRS quadrant framework in the report sums up our wide spectrum of data-driven research and advisory for CXOs to help them make better decisions for their businesses and stay as leaders.

Below is a snapshot of these quadrants.

1. Customer Experience Map

The study offers an in-depth assessment of various customers’ journeys pertinent to the market and its segments. It offers various customer impressions about the products and service use. The analysis takes a closer look at their pain points and fears across various customer touchpoints. The consultation and business intelligence solutions will help interested stakeholders, including CXOs, define customer experience maps tailored to their needs. This will help them aim at boosting customer engagement with their brands.

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The findings presented in this study by TMR are an indispensable guide for meeting all business priorities, including mission-critical ones. The results when implemented have shown tangible benefits to business stakeholders and industry entities to boost their performance. The results are tailored to fit the individual strategic framework. The study also illustrates some of the recent case studies on solving various problems by companies they faced in their consolidation journey.

4. Strategic Frameworks

The study equips businesses and anyone interested in the market to frame broad strategic frameworks. This has become more important than ever, given the current uncertainty due to COVID-19. The study deliberates on consultations to overcome various such past disruptions and foresees new ones to boost the preparedness. The frameworks help businesses plan their strategic alignments for recovery from such disruptive trends. Further, analysts at TMR helps you break down the complex scenario and bring resiliency in uncertain times.

The report sheds light on various aspects and answers pertinent questions on the market. Some of the important ones are:

1. What can be the best investment choices for venturing into new product and service lines?

2. What value propositions should businesses aim at while making new research and development funding?

3. Which regulations will be most helpful for stakeholders to boost their supply chain network?

4. Which regions might see the demand maturing in certain segments in near future?

5. What are the some of the best cost optimization strategies with vendors that some well-entrenched players have gained success with?

6. Which are the key perspectives that the C-suite are leveraging to move businesses to new growth trajectory?

7. Which government regulations might challenge the status of key regional markets?

8. How will the emerging political and economic scenario affect opportunities in key growth areas?

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10. What will be the barrier to entry for new players in the market?

Note: Although care has been taken to maintain the highest levels of accuracy in TMR’s reports, recent market/vendor-specific changes may take time to reflect in the analysis.

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Neurometabolic Disorders Market

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