Sanfilippo Syndrome Market: Breakthrough Forecast Study Predicts Astounding Growth in the Upcoming Years | Seelos Therapeutics, Lysogene, BioMarin Pharmaceutical, Orchard Therapeutics, ArmaGen

As per DelveInsight, the Sanfilippo Syndrome Market is anticipated to evolve immensely in the coming years owing to the improvement in the diagnosis methodologies, raising awareness of the diseases, incremental healthcare spending across the world, and the expected launch of emerging therapies.

DelveInsight's "Sanfilippo Syndrome Market Insights, Epidemiology, and Market Forecast 2032" report delivers an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the Sanfilippo Syndrome Market Size and Share in the seven major markets (7MM) (i.e., the United States, EU4 (Germany, Spain, Italy, France), the United Kingdom and Japan).

The market report covers emerging drugs, treatment practices, market share of individual Sanfilippo Syndrome therapies, and current & forecasted market size from 2019 to 2032. It also evaluates the current Sanfilippo Syndrome treatment practice/algorithm, market drivers & barriers, and unmet medical needs to curate the best of opportunities and assess the underlying potential of the market.

Sanfilippo Syndrome: An Overview
Sanfilippo Syndrome, also known as Mucopolysaccharidosis type III (MPSIII), is a rare genetic condition that causes fatal brain damage. It is referred to as a childhood disease because most patients fail to reach adulthood. Sanfilippo is a metabolic disorder. One in 70,000 children is born with this inherited condition, which is caused by an enzyme deficiency.

The lack of enzymes prevents the body from going through its natural recycling process, causing cellular malfunction. The disease has four subtypes (A, B, C, and D). Each subtype corresponds to a specific deficient enzyme.

Sanfilippo affects mostly the Central Nervous System (CNS). Over time, brain cells fill up with waste that the body is unable to process. As the brain gets progressively damaged, children experience hyperactivity, sleeplessness, loss of speech, cognitive skills, mental retardation, cardiac issues, seizures, loss of mobility, dementia, and finally death, usually before adulthood. Since the genetic mutations are located in the autosomes and not in the sex chromosomes; therefore, Sanfilippo syndrome affects males and females equally.

Sanfilippo Syndrome Market Key Facts
• According to the National Organization for Rare Disorders, MPS III has been classified as a rare disease with incidence reported to be between 0.28 and 4.1 cases per 100,000 births.
• MPS IIIA is the most common subtype, affecting around 1 in 100,000 births, closely followed by type B at 1 in 200,000. In some countries in Southern Europe, type B has been reported to be more common than A.
• MPS IIIC and IIID are rarer, with reported incidences of approximately 1 in 1.5 million and 1 in 1 million births, respectively.

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Sanfilippo Syndrome Market
The market outlook section of the report helps to build a detailed comprehension of the historical, current, and forecasted market size by analyzing the impact of current and emerging Sanfilippo Syndrome therapies in the market. It also provides a detailed assessment of the Sanfilippo Syndrome market drivers & barriers, unmet needs, and emerging technologies.

The report gives complete details of the market trend for each marketed Sanfilippo Syndrome drug and late-stage pipeline therapies by evaluating their impact based on the annual cost of therapy, inclusion and exclusion criteria, mechanism of action (MOA), competition with other therapies, brand value, their impact on the market and view of the key opinion leaders.

Sanfilippo Syndrome Epidemiology
The epidemiology section covers detailed insights into the historical, and current Sanfilippo Syndrome patient pool and forecasted epidemiology trends for every seven major countries (7MM) from 2019 to 2032.

The Report Covers the Sanfilippo Syndrome Epidemiology Segmented as -
• Total Prevalent Cases of Sanfilippo Syndrome (2019-2032)
• Sub-type Specific Prevalence of Sanfilippo Syndrome (2019-2032)
• Diagnosed and Treatable Population of Sanfilippo Syndrome (2019-2032)

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Sanfilippo Syndrome Drugs Uptake and Pipeline Development Activities
The drugs' uptake section focuses on the rate of uptake of the potential drugs recently launched in the Sanfilippo Syndrome market or expected to be launched during the study period. The analysis covers the market share of Sanfilippo Syndrome drugs, patient uptake by therapies, and sales of each drug.

The report's drugs uptake section helps in understanding the drugs with the most rapid uptake and the reasons behind the maximal use of new drugs. It allows the comparison of the drugs based on market share and size, which again will be helpful in investigating factors important in understanding the market dynamics and in making financial and regulatory decisions.

The report also covers the Sanfilippo Syndrome 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 analyzes recent developments such as collaborations, acquisitions, mergers, licensing patent details, and other information for emerging therapies.

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Sanfilippo Syndrome Therapeutics Assessment
Hematopoietic stem cell transplantation is one of the possible treatments available for MPS, which has been previously used in Hurler patients. Such transplants have been performed in patients with MPS IIIA and B; however, the neurological benefits have been widely questioned and hence this is not currently considered as an effective option. On the other hand, there have recently been carried out clinical trials of enzyme replacement therapy (ERT) using recombinant enzymes. ERT is currently the most successful treatment for the non-neurological symptoms of lysosomal storage diseases in general.

Other therapeutic approaches include the use of pharmacological chaperones, which are specific molecules capable of increasing residual lysosomal enzyme activity by rescuing unfolded mutated enzymes from the endoplasmic reticulum.

Gene therapy which transfers the wild-type gene to correct the genetic defect may be the best option,
though most complex. The main challenge with this therapy is to achieve the insertion of the normal gene into hundreds of millions of cells that contain the defective gene; for this, a carrier is needed to transport the gene inside the cells.

Currently, there is no approved therapy for the treatment of Sanfilippo syndrome. However, several major pharma and biotech companies are actively working in the therapeutics market.

Leading Companies in the Sanfilippo Syndrome Therapeutics Market Include
• Seelos Therapeutics
• Lysogene
• BioMarin Pharmaceutical
• Orchard therapeutics
• ArmaGen
And Many More

Emerging and Marketed Sanfilippo Syndrome Therapies Covered in the Report Include
• SLS-005: Seelos Therapeutics
• LYS-SAF302: Lysogene
• Tralesinidase alfa (BMN-250): BioMarin Pharmaceutical
• OTL-201: Orchard therapeutics
• AGT-184: ArmaGen
And Many Others

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Table of Content (TOC)
1. Key Insights
2. Executive Summary
3. Sanfilippo Syndrome Competitive Intelligence Analysis
4. Sanfilippo Syndrome Market Overview at a Glance
5. Sanfilippo Syndrome Background and Overview
6. Sanfilippo Syndrome Patient Journey
7. Sanfilippo Syndrome Epidemiology and Patient Population
8. Sanfilippo Syndrome Treatment Algorithm, Current Treatment, and Medical Practices
9. Sanfilippo Syndrome Unmet Needs
10. Key Endpoints of Sanfilippo Syndrome Treatment
11. Sanfilippo Syndrome Marketed Products
12. Sanfilippo Syndrome Emerging Therapies
13. Sanfilippo Syndrome Seven Major Market Analysis
14. Attribute Analysis
15. Sanfilippo Syndrome Market Outlook (7 major markets)
16. Sanfilippo Syndrome Access and Reimbursement Overview
17. KOL Views on the Sanfilippo Syndrome Market.
18. Sanfilippo Syndrome Market Drivers
19. Sanfilippo Syndrome Market Barriers
20. Appendix
21. DelveInsight Capabilities
22. Disclaimer
*The table of contents is not exhaustive; the final content may vary.

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Email: info@delveinsight.com

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About DelveInsight
DelveInsight is a leading Business Consultant and Market Research firm focused exclusively on life sciences. It supports Pharma companies by providing comprehensive end-to-end solutions to improve their performance.

This release was published on openPR.