Acid Sphingomyelinase Deficiency Market Report gives readers a thorough assessment of key market drivers, restraints, prospects, opportunities, restraints, current trends, and technological and industrial breakthroughs that will influence market growth. The detailed market research, industry sector growth and development, and new product introductions covered in this report on the Acid Sphingomyelinase Deficiency Market are of great assistance to the important new commercial players joining the market.

 

This Acid Sphingomyelinase Deficiency Market report research conducts a careful market evaluation and provides a professional analysis of the industry taking into account market development, present market circumstances, and forecasts for the future. The market driving factors, market overview, industry volume, and market share are also highlighted in this Acid Sphingomyelinase Deficiency Market report analysis.

 

Regions Covered in the Report-

• The United States
• EU5 (Germany, France, Italy, Spain, and the United Kingdom)
• Japan

 

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Acid Sphingomyelinase Deficiency: An Overview

Acid sphingomyelinase deficiency (ASMD) is a rare progressive genetic disorder, caused by the mutations in the SMPD1 gene and is inherited in an autosomal recessive manner. Any mutation in the gene, leads to the production of a faulty protein and deficiency of the enzyme acidsphingomyelinase, required to break down a fatty substance called sphingomyelin. Consequently, sphingomyelin and other substances accumulate in various tissues of the body and can affect many organ of the system including brain, liver, spleen, lung etc.

 

Key highlights of the Acid Sphingomyelinase Deficiency Market Report

• As per the study by the McGovern et al. (2017), titled “Disease manifestations and burden of illness in patients with acid sphingomyelinase deficiency (ASMD)”, it was reported that the incidence of ASMD is 0.5 per 100,000 births, The study also reported that estimates extrapolated from the results of carrier screening suggest that the true incidence may be higher in select populations.
• 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.
• A study by Escobar et al. titled “Niemann-Pick Disease: An Approach for Diagnosis in Adulthood” (2019), quoted that NPD is diagnosed in 80% of cases under the age of 16 years, in 14% after 20 years of age, and extremely rare in older age, depending on the subtype.

 

Acid Sphingomyelinase Deficiency Epidemiology Insights

ASMD has traditionally been broken down into two subgroups – neuronopathic (type A) and non-neuronopathic (type B). Neuronopathic refers to disorders that damage brain cells (neurons). Type A generally causes severe neurodegenerative disease during infancy, while type B is generally not considered to be a neurologic disease. However, since cases fall in between these two extremes, an intermediate form, type A/B can also be seen. The main cause of ASMD is mutation in the sphingomyelin phosphodiesterase-1 (SMPD1) gene. Genes provide instructions for creating proteins that play a critical role in many body functions. When a gene mutation occurs, the protein product may be faulty, inefficient, or absent. Depending upon the functions of the particular protein, this can affect many organ systems of the body, including the brain.

 

Acid Sphingomyelinase Deficiency Epidemiology Segmentation in the 7MM

• Total Treated Cases
• Total Diagnosed Prevalence Cases

 

Acid Sphingomyelinase Deficiency Treatment Market

There is a tremendous lack of effective supportive therapies (especially for the infantile cases), let alone curative ones with the severe infantile neurovisceral phenotype encountering death by 3 years of age. Although the other less severe forms have a normal lifespan but a certain section die prematurely due to respiratory insufficiency and liver disease. Statins form the mainstay of treatment regimen that is used to treat hyperlipidemia characterized by low-high-density lipoprotein (HDL) cholesterol in NPD Type B adult cases.

 

Acid Sphingomyelinase Deficiency Market Insights

NPD B and A/B is associated with a substantial burden for many patients, caused by the profoundly negative impact of its clinical manifestations on physical, mental and psychosocial well-being. However, published data regarding the impact of the disease on QoL and daily functioning are limited, mainly owing to the rarity and heterogeneity of the disease and the lack of adequate disease-specific instruments to measure QoL. Given the wide spectrum of NPD B phenotypes, including intermediate forms characterized by mild to severe neurologic defects in addition to visceral, skeletal and hematologic ASMD manifestations, a refinement of the ASMD classification system is needed. A more precise description of ASMD subpopulations may improve disease recognition at initial presentation and thus lead to more timely diagnoses.

 

Acid Sphingomyelinase Deficiency Emerging Therapies

• Xenpozyme (Olipudase Alfa): Sanofi Genzyme (Japan)
• Olipudase Alfa: Sanofi Genzyme (US, EU)

 

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Acid Sphingomyelinase Deficiency Market Drivers

• Incentives for rare diseases
• Lack of competition in the ASMD market

 

Acid Sphingomyelinase Deficiency Market Barriers

• Lack of patients in clinical trials
• High cost of research

 

Table of Content

1. Key Insights

2. Executive Summary

3. Competitive Intelligence Analysis for Acid sphingomyelinase deficiency (ASMD)

4. Acid sphingomyelinase deficiency (ASMD): Market Overview at a Glance

5. Acid sphingomyelinase deficiency (ASMD): Disease Background and Overview

6. Patient Journey

7. Acid sphingomyelinase deficiency (ASMD) Epidemiology and Patient Population

8. Treatment Algorithm, Current Treatment, and Medical Practices

9. Acid sphingomyelinase deficiency (ASMD) Unmet Needs

10. Key Endpoints of Acid sphingomyelinase deficiency (ASMD) Treatment

11. Acid sphingomyelinase deficiency (ASMD) Marketed Products

12. Acid sphingomyelinase deficiency (ASMD) Emerging Therapies

13. Acid sphingomyelinase deficiency (ASMD): Seven Major Market Analysis

14. Attribute analysis

15. 7MM: Acid Sphingomyelinase Deficiency Market Outlook

16. Access and Reimbursement Overview of Acid sphingomyelinase deficiency (ASMD)

17. KOL Views

18. Acid Sphingomyelinase Deficiency Market Drivers

19. Acid Sphingomyelinase Deficiency Market Barriers

20. Appendix

21. DelveInsight Capabilities

22. Disclaimer

23. About DelveInsight

 

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