DelveInsight Business Research LLP
Albany, NY -- (SBWIRE) -- 03/26/2020 -- Sanfilippo Syndrome Market Insights, Epidemiology and Market Forecast-2030
(Albany, US) DelveInsight launched a new report on Sanfilippo Syndrome Market Insights, Epidemiology and Market Forecast-2030
Some of the key facts of the report
1. One in 70,000 children is born with Sanfilippo Syndrome, which is caused by an enzyme deficiency.
2. Sanfilippo Syndrome or Mucopolysaccharidosis type III (MPS III), has been classified as a rare disease with an incidence reported to be between 0.28 and 4.1 cases per 100,000 births.
3. MPS III A is the most common subtype that affects around 1 in 100,000 births.
Key benefits of the report
1. Sanfilippo Syndrome market report covers a descriptive overview and comprehensive insight of the Sanfilippo Syndrome epidemiology and Sanfilippo Syndrome market in the 7 MM (the United States, EU5 (Germany, Spain, France, Italy, UK) & Japan.)
2. Sanfilippo Syndrome market report provides insights on the current and emerging therapies.
3. Sanfilippo Syndrome market report provides a global historical and forecasted market covering drug outreach in 7 MM.
4. Sanfilippo Syndrome market report offers an edge that will help in developing business strategies by understanding trends shaping and driving the Sanfilippo Syndrome market.
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"Sanfilippo syndrome affects males and females equally."
Conventionally, Sanfilippo syndrome treatments are based on different approaches like Multidisciplinary support treatment, Deficient enzyme replacement, either with external administration by hematopoietic stem cell or chaperones transplantation that stabilizes the protein, Reduction of substrate accumulation by inhibiting the enzymes synthesizing it, and Gene therapy.
Hematopoietic stem cell transplantation is one of the possible Mucopolysaccharidosis treatments available, 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 a practical 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 transfers the wild-type gene to correct the genetic defect, maybe the best option, though most complex. The main problem 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.
There is no, currently, recommended therapy for the treatment of Sanfilippo syndrome. The neurodegenerative nature of mucopolysaccharidosis type III (MPS III) and the difficulty for drugs to cross the blood-brain barrier, has determined that it is proving challenging to have an effective therapy for these patients. However, at present, several therapies focused on the central nervous system are undergoing testing. In the coming years, the treatment for patients with MPS III will have a significant change with the coming of new therapies. At the moment, clinical trials with enzyme replacement therapy for MPS IIIA (intrathecal) and MPS IIIB (intravenous), as well as the extension study of patients with intracerebral gene therapy, are being conducted. It is expected that soon, trials with intravenous and intraventricular gene therapy will be initiated in patients with MPS IIIA and MPS IIIB. It will have a positive impact on the Sanfilippo syndrome market size in the upcoming years.
The launch of the emerging therapies is expected to significantly impact Sanfilippo Syndrome treatment scenario in the upcoming years:-
3. Tralesinidase alfa (BMN-250)
And many others
The key players in Sanfilippo Syndrome market are:
1. Seelos Therapeutics
3. BioMarin Pharmaceutical
And many others
Table of contents
1. Report Introduction
2. Sanfilippo syndrome Market Overview at a Glance
3. Sanfilippo syndrome Disease Background and Overview
4. Sanfilippo syndrome Epidemiology and Patient Population
5. Sanfilippo syndrome Epidemiology by Countries (2017-2030)
5.1. United States- Epidemiology (2017-2030)
5.2. EU-5 - Epidemiology (2017-2030)
5.3. Germany-Epidemiology (2017-2030)
5.4. France-Epidemiology (2017-2030)
5.5. Italy-Epidemiology (2017-2030)
5.6. Spain-Epidemiology (2017-2030)
5.8. Japan-Epidemiology (2017-2030)
6. Sanfilippo syndrome treatments & Medical Practices
7. Sanfilippo syndrome Emerging Therapies
7.1. Key Cross Competition
7.2. SLS-005:Seelos Therapeutics
7.3. LYS-SAF302: Lysogene
7.4. Tralesinidase alfa (BMN-250): BioMarin Pharmaceutical
8. Sanfilippo syndrome Market Size
9. 7MM: Country-Wise Market Analysis
9.1. United States Market Size
9.2. Germany Market Size
9.3. France Market Size
9.4. Italy Market Size
9.5. Spain Market Size
9.6. United Kingdom Market Size
9.7. Japan Market Size
10. Market Drivers
11. Market Barriers
12. Sanfilippo syndrome Report Methodology
13. DelveInsight Capabilities
15. About DelveInsight
DelveInsight is a leading Business Consultant, and Market Research Firm focused exclusively on life sciences. It supports pharma companies by providing end to end comprehensive solutions to improve their performance.