Acid sphingomyelinase (aSMases) is one of the enzymes from the family sphingomyelinase (SMase). It is an enzyme responsible for the development of phosphorylcholine and ceramide molecules through the breakdown of sphingomyelin. Acid sphingomyelinase are organized into acidic, neutral, and alkaline SMase based on the pH range in which their enzymatic action is optimal. The enzymatic activity can be affected by redox proteins, pH, cations, lipids, and several other proteins. Enzymatic activity of aSMases increases in phosphatidylinositol (PI) or lysobisphophatidic acid (LBPA) improved environments, and its activity reduces when phosphorylated derivatives of phosphatidylinositol are present in the surroundings. Acid sphingomyelinase are of two types: secretory sphingomyelinase (S-SMase) and lysosomal sphingomyelinase (L-SMase). Sphingomyelin phosphodiesterase 1 (SMPD1) is a gene coded for two aSMase enzymes from different groups of sphingomyelin these hydrolyse. Lysosomal sphingomyelinase (L-SMase) is present in the lysosomal compartment and secretory sphingomyelinase (S-SMase) is present at extracellular portion.

Acid sphingomyelinase deficiency is also called ASM-deficient Niemann-Pick disease, acid sphingomyelinase-deficient Niemann-Pick disease, ASM deficiency, or ASMD. It is an uncommon progressive genetic disorder caused by insufficiency of the enzyme acid sphingomyelinase, which is necessary to breakdown lipid (fatty) material known as sphingomyelin. Therefore, sphingomyelin and other elements gather in several tissues of the body. ASM deficiency is extremely flexible and the age of onset, severity, and definite symptoms of the disorder can differ dramatically from one person to another, occasionally even between members of the same family.

Acid sphingomyelinase deficiency disorder can be considered a spectrum of disease. At the severe side of the spectrum, it causes fatal neurodegenerative disorder that presents in the beginning. At the minor end of the spectrum, it affects people with no symptoms or only negligible neurological symptoms and survival into adulthood is common. There is another form of disorder that exists as intermediate condition. ASM deficiency is caused by the mutation in SMPD1 gene and is inherited in an autosomal recessive manner. The global acid sphingomyelinase deficiency market is driven by increase in genetic mutations due to radiation and several other factors. Unavailability of medication and unawareness about the disorder are the major factors restraining the global market.

The global acid sphingomyelinase deficiency market can be segmented based on product, end-user, and region. In terms of product, the market can be classified into Olipudase Alfa, OKL-1014, LJPC-0712, ML-SA1, OR-0005, and others. In terms of end-user, the global acid sphingomyelinase deficiency market can be divided into hospitals, clinics, and others.

In terms of region, the global acid sphingomyelinase deficiency market can be segmented into North America, Europe, Asia Pacific, Latin America, and Middle East & Africa. North America is expected to dominate the global market due to availability of advanced technology and treatment. Awareness among the people in the U.S. is also a major driver of the market in the region.

Increase in government initiatives to reduce the incidence rate of the disorder and surge in health care expenditure are the major factors propelling the acid sphingomyelinase deficiency market is Asia Pacific. Lack of awareness about acid sphingomyelinase deficiency disorders and unavailability of advanced technology and treatment in underdeveloped countries such as Turkey, Cyprus, Syria, Lebanon, and Iraq are likely to propel the acid sphingomyelinase deficiency market in Middle East & Africa. Increase in demand for acid sphingomyelinase deficiency treatment offers significant opportunities for players in the market in these regions.

Top players in the global acid sphingomyelinase deficiency market are Orphazyme ApS, Okklo Life Sciences BV, Merck & Co., Inc., Genzyme Corporation, and La Jolla Pharmaceutical Company, among others.

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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Acid Sphingomyelinase Deficiency Market

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