The Acid Sphingomyelinase Deficiency Market Research Report covers emerging drugs, current treatment practices, market share of the individual therapies, current and forecasted Acid Sphingomyelinase Deficiency market Size from 2017 to 2030 segmented by seven major markets.
The report provides a detailed current Acid Sphingomyelinase Deficiency treatment practice/algorithm, market drivers, market barriers, and unmet medical needs to curate the best of the opportunities and assess the underlying potential of the market.
Acid sphingomyelinase deficiency (ASMD) is a rare progressive genetic disorder that results from a deficiency of the enzyme acid sphingomyelinase, which is required to break down (metabolize) a fatty substance (lipid) called sphingomyelin that is inherited in an autosomal recessive pattern.
The primary organ systems affected in all ASM-deficient patients are the spleen, liver, and lung. The onset and severity of pulmonary disease in ASM-deficient NPD are highly variable and are primarily due to the infiltration of inflammatory cells into the airways. It is notable that many patients exhibit joint and bone pain, and there may be a higher incidence of fractures.
According to the National Organization of Rare Diseases, the exact incidence and prevalence of the disorder is unknown but has been estimated at 1 in 250,000 individuals in the general population.
As per the study published by Margaret et al., the incidence of ASMD at approximately 0.5 per 100,000 births, estimates extrapolated from the results of carrier screening, which suggest that the true incidence may be higher in selected populations.
According to the study published by Giugliani et al., ASMD has an autosomal recessive pattern of inheritance, and the birth prevalence is estimated at 0.4–0.6 per 100,000.
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Key Benefits of Acid Sphingomyelinase Deficiency Market Report
The market report provides an in-depth analysis of Acid Sphingomyelinase Deficiency Market Size, Share, Trend, and Epidemiology Forecast till 2030 in the seven major markets (i.e. the United States, EU5 (Germany, Italy, Spain, France, and the UK) and Japan.
The report will help in developing business strategies by understanding the Acid Sphingomyelinase Deficiency Market Trends, recent developments, key players, and future market competition that will shape and drive the market growth in the upcoming years.
The market report covers the Acid Sphingomyelinase Deficiency current treatment practices, emerging drugs, market share of the individual therapies in the seven major markets.
The report provides a detailed assessment of the Acid Sphingomyelinase Deficiency market in terms of market drivers & barriers, unmet needs, market opportunities, patient population, detailed comparative analysis of pipeline products including clinical and non-clinical stage products, and other factors.
Acid Sphingomyelinase Deficiency Market
Owing to the lack of any approved therapeutic products the Acid Sphingomyelinase Deficiency (ASMD) market is yet to address its high unmet need.
The market outlook section of the report helps to build a detailed comprehension of the historic, current, and forecasted Acid Sphingomyelinase Deficiency market size and share by analyzing the impact of current therapies on the market, unmet needs, drivers, and barriers, and demand for better technology.
The report gives a thorough detail of the Acid Sphingomyelinase Deficiency market trend of each marketed drug and late-stage pipeline therapy by evaluating their impact based on the annual cost of therapy, inclusion and exclusion criteria, mechanism of action, increasing patient pool, covered patient segment, expected launch year, competition with other therapies, brand value, their impact on the market and view of the key opinion leaders.
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Acid Sphingomyelinase Deficiency Epidemiology
The epidemiology section covers insights about the historical and current Acid Sphingomyelinase Deficiency patient pool and forecasted trends for every seven major countries from 2017 to 2030. It helps to recognize the causes of current and forecasted trends by exploring numerous studies and views of key opinion leaders. The epidemiology section also provides the diagnosed patient pool and their trends along with assumptions undertaken.
Acid Sphingomyelinase Deficiency (ASMD) Epidemiology Segmentation-
Total Prevalent cases of Acid sphingomyelinase deficiency
Diagnosed cases of Acid sphingomyelinase deficiency (ASMD)
Diagnosed Cases of Acid sphingomyelinase deficiency (ASMD) by clinical phenotype
Diagnosed Cases of Acid sphingomyelinase deficiency (ASMD) based on clinical manifestations
Acid Sphingomyelinase Deficiency Drugs Uptake and Pipeline Development Activities
The drugs uptake section focuses on the rate of uptake of the potential drugs recently launched in the Acid Sphingomyelinase Deficiency market or expected to get launched in the market during the study period. The analysis covers Acid Sphingomyelinase Deficiency market uptake by drugs; patient uptake by therapies; and sales of each drug.
Report’s Drugs Uptake section helps in understanding the drugs with the most rapid uptake, reasons behind the maximal use of new drugs and allow the comparison of the drugs on the basis of market share and size which again will be useful in investigating factors important in market uptake and in making financial and regulatory decisions.
The report also covers the Acid Sphingomyelinase Deficiency Pipeline Development Activities and provides valuable insights about different therapeutic candidates in Phase II, and Phase III stages and the key companies in the market involved in developing targeted therapeutics. It also analyses the recent Development such as collaborations, acquisitions, and mergers, licensing patent details, and other information for emerging therapies.
There is only one company, i.e., Sanofi (Genzyme) which is currently engaged in developing a therapy for the treatment of Acid Sphingomyelinase Deficiency. Sanofi is workig on Olipudase Alfa.
Olipudase Alpha is recombinant human acid sphingomyelinase-based ERT that targets the non-neurological manifestations of acid sphingomyelinase deficiency (ASMD), also known as Niemann–Pick B disease. It has received both orphan drug designation and breakthrough therapy designation from the USFDA.
Since Sanofi is the only company that is currently working to develop a therapy for the treatment of ASMD, it presents an opportunity for the company to capture the highly available untapped market due to the unavailability of any approved treatment for ASMD.
Table of Content
1. Key Insights
2. Executive Summary
3. Acid Sphingomyelinase Deficiency Competitive Intelligence Analysis
4. Acid Sphingomyelinase Deficiency Market Overview at a Glance
5. Acid Sphingomyelinase Deficiency Disease Background and Overview
6. Acid Sphingomyelinase Deficiency Patient Journey
7. Acid Sphingomyelinase Deficiency Epidemiology and Patient Population
8. Acid Sphingomyelinase Deficiency Treatment Algorithm, Current Treatment, and Medical Practices
9. Acid Sphingomyelinase Deficiency Unmet Needs
10. Key Endpoints of Acid Sphingomyelinase Deficiency Treatment
11. Acid Sphingomyelinase Deficiency Marketed Products
12. Acid Sphingomyelinase Deficiency Emerging Therapies
13. Acid Sphingomyelinase Deficiency Seven Major Market Analysis
14. Attribute Analysis
15. Acid Sphingomyelinase Deficiency Market Outlook (7 major markets)
16. Acid Sphingomyelinase Deficiency Access and Reimbursement Overview
17. KOL Views on the Acid Sphingomyelinase Deficiency Market.
18. Acid Sphingomyelinase Deficiency Market Drivers
19. Acid Sphingomyelinase Deficiency Market Barriers
21. DelveInsight Capabilities
*The table of contents is not exhaustive; the final content may vary.
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