Hurler syndrome or mucopolysaccharidosis I (MPS I) is an uncommon hereditary issue ailment of digestion in which a human body can't separate long chains of sugar particles called glycosaminoglycansdue due to an insufficiency of alpha-L-iduronidase, an enzyme responsible for the degradation of mucopolysaccharides in lysosomes. Moreover, without the alpha-L-iduronidase catalyst, a development of heparan sulfate and dermatan sulfate takes place in the body. Symptoms of Hurler syndrome appear in the early stage of life (childhood) and can lead to death due to organ harm or damage. Symptoms of Hurler syndrome appear between 3 years and 8 years. Infants with severe Hurler syndrome seem normal at birth. Facial signs and symptoms become more clear and visible during the first two years of life. Hurler syndrome is marked by progressive weakening, hepatosplenomegaly, dwarfism, and unique facial features. There is progressive mental retardation, with death frequently occurring by the age of 10.
The mucopolysaccharidosis I (MPS I) or Hurler syndrome market has been segmented based on diagnosis, treatment, and region. In terms of diagnosis, the market has been categorized into blood test, urine test, amniocentesis, genetic testing, and others. The blood test segment held the largest share of the market due to easy access for outpatient department (OPD) patients to carry out tests. The genetic testing segment is likely to witness strong growth during the forecast period due to increasing awareness about mucopolysaccharidosis I (MPS I) or Hurler syndrome. In terms of treatment, the market has been categorized into enzyme replacement therapy, bone marrow transplantation, surgery, and others. The enzyme replacement therapy segment holds the largest share of the mucopolysaccharidosis I (MPS I) or Hurler syndrome market due to increasing incidence of syndrome globally. The bone marrow transplantation (BMT) segment is likely to witness significant growth in the next few years driven by effective treatment of the syndrome.
Geographically, the mucopolysaccharidosis I (MPS I) or Hurler syndrome market has been segmented into five major regions: North America (U.S. and Canada), Europe (Germany, France, U.K., etc.), Asia Pacific (Australia, China, Japan, India, etc.), Latin America (Brazil, Mexico, etc.), and Middle East &Africa (GCC Countries, South Africa, etc.). North America is the leading market for mucopolysaccharidosis I (MPS I) or Hurler syndrome in terms of revenue, followed by Europe. The U.S. holds the largest share of the market in the region. Asia Pacific and Latin America are emerging markets, which are expected to witness strong growth during the forecast period from 2017 to 2025. Strong growth in these regions is attributed to increasing incidence of syndrome, rising health care industries, and increase in government investment to develop health care infrastructure. The mucopolysaccharidosis I (MPS I) or Hurler syndrome market in countries such as China, India, and Brazil is projected to expand at high growth rate during the forecast period due to increase in awareness among the population about the syndrome and its treatment.
Other factors that drive the mucopolysaccharidosis I (MPS I) or Hurler syndrome market are rising prevalence of syndrome, increasing novel drug treatment, high adoption and acceptance in developing markets, and innovations and developments in treatment. The market is also experiencing certain restraints such as high possibilities of substance abuse and addiction, complications and severity involved in treatment, high cost of treatment, and lack of awareness among the rural population in developing and underdeveloped economies.
The treatment regime for mucopolysaccharidosis I (MPS I) or Hurler syndrome is determined depending on the severity of the symptoms and varies from patient to patient and case to case. Since there is no permanent cure for the syndrome, treatment primarily focuses on management of signs, indications, and complications associated with the disease progression. Palliative care is given to manage various conditions such as heart complications, respiratory complications, behavioral and neurological complications, sleep issues, and skeletal and connective tissue problems.
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
- 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.