Lysosomal storage disease (LTD) is caused by defect in lysosomal functions and degradation pathway. It is caused by progressive accumulation of metabolites. LTD is a rare inherited metabolic disorder. There are 50 types of lysosomal storage diseases. Lysosomes are organelle present in eukaryotic cell. These consist of degradative enzymes enclosed in a membrane. A study conducted by Mayo Clinic in the U.S. indicated that the incidence of lysosomal disorder ranges between 1 in 4000 and 1 in 13,000 live births. Tay-Sachs disease was the first lysosomal disorder to be diagnosed in 1881.

Increase in incidence of lysosomal diseases, rise in diagnostic rate, surge in awareness about this rare disorder, increase in focus on research and development for diagnosis & drug development for treatment of lysosomal diseases, and implementation of orphan drug regulation that provides marketing and commercial benefits to pharmaceutical companies fuel the growth of the global lysosomal storage disease treatment market. However, high cost of available treatment, underdiagnosed lysosomal disorders because of heterogeneity of the rare lysosomal disorder, and decrease in efficiency of treatment option are likely to hamper the growth of the global lysosomal storage disease treatment market. Availability and advancement of chaperone and gene therapy is expected to propel the market during the forecast period.

The global lysosomal storage disease treatment market can be segmented based on disease type, therapy, end-user, and region. In terms of disease type, the global market can be classified into aspartylglucosaminuria, Batten disease, cystinosis, Fabry disease, Gaucher disease, glycogen storage disease II, Tay-Sachs disease, and others. Gaucher disease is the most common type of lysosomal disorder which is characterized by neurological complication. Tay-Sachs disease is another common lysosomal disorder propelling the global lysosomal storage disease treatment market.
Based on therapy, the global lysosomal storage disease treatment market can be categorized into enzyme replacement therapy, stem cell transplantation, substrate reduction therapy, and others. Enzyme replacement is the most preferred therapy for the treatment of lysosomal disorder. It is effective in reducing toxic storage material and organomegaly. Enzyme replacement therapy also helps in improving organ function in patients suffering from lysosomal disorder. Advanced research in stem cell transplantation has increased the success rate of treatment of lysosomal disease. Rise in the number of stem cell banks also fuels the growth of the global lysosomal storage disease treatment market.

In terms of end-user, the global lysosomal storage disease treatment market can be divided into hospitals, clinics, stem transplant centers, research organizations, and others. Hospitals was the leading segment of the global lysosomal storage disease treatment market in 2017. Research organizations are being funded by governments as well as pharmaceutical companies for the development of new treatment methods and drugs. Hence, the research organizations segment is expected to witness significant growth during the forecast period.

In terms of region, the global lysosomal storage disease treatment market can be segmented into North America, Europe, Asia Pacific, Latin America, and Middle East & Africa. North America is a leading market for lysosomal disease treatment because of strong health care, economic, and financial infrastructure. Europe is a major hub for health care research and development. Moreover, high per capita income of the people is attributed to the growth of the global lysosomal storage disease disorder treatment market in the region. Asia Pacific consists of developing countries with high prevalence of inherited abnormalities and disorders. Hence, the market in the region is expected to grow at a rapid pace during the forecast period.

Key players operating in the global lysosomal storage disease treatment market include Shire, Pfizer, Inc., Sanofi, BioMarin, Merck & Co., Inc., Actelion Pharmaceuticals Ltd., and Eli Lilly and Company.

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
  • Insights and Tools based on data-driven research
  • Actionable Results to meet all the business priorities
  • 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.

2. Insights and Tools

The various insights in the study are based on elaborate cycles of primary and secondary research the analysts engage with during the course of research. The analysts and expert advisors at TMR adopt industry-wide, quantitative customer insights tools and market projection methodologies to arrive at results, which makes them reliable. The study not just offers estimations and projections, but also an uncluttered evaluation of these figures on the market dynamics. These insights merge data-driven research framework with qualitative consultations for business owners, CXOs, policy makers, and investors. The insights will also help their customers overcome their fears.

3. Actionable Results

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?

9. What are some of the value-grab opportunities in various segments?

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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Lysosomal Storage Disease Treatment Market

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