DelveInsight has launched a new report on “Sanfilippo Syndrome Pipeline”
Sanfilippo syndrome, or mucopolysaccharidosis type III (MPS III), is a disorder of metabolism, associated with insufficient production of a lysosomal enzyme needed for normal cell function. As a consequence of the cellular dysfunction, patients with this disorder develop progressive, irreversible neurodegeneration.
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Sanfilippo Syndrome Pipeline analysis across different stages of development (Phase I, II, and III), different emerging trends and comparative analysis of pipeline products with detailed clinical profiles, key competitors, launch date along with product development activities.
Sanfilippo Syndrome Pipeline
Genomic DNA sequencing is achieved by employing oligonucleotide primers that span the intron/exon junctions of the exons of the gene of interest using the polymerase chain reaction with genomic DNA as the template. Products can be sequenced and cross-referenced to the standard wild-type sequence and known disease-causing mutations and polymorphisms.
There is currently no therapy available for MPS III. Animal models exist for all forms, whether spontaneous or generated via gene targeting. These have contributed to a better understanding of MPS III, and will continue to deliver invaluable tools to evaluate emerging therapies.
Inflammation has been connected with disease pathogenesis in the MPS disorders. Therapies aimed at decreasing inflammation are currently being studied in many MPS disorders and benefits in both brain and other parts of the body have been reported.
Emerging therapies included in the report
Sanfilippo Syndrome Emerging Therapy
Allievex has obtained an exclusive, worldwide license from BioMarin Pharmaceutical under which Allievex would assume all financial obligations associated with the development and commercialization of tralesinidase alfa, while BioMarin would provide certain manufacturing services in support of a marketing authorization filing. Tralesinidase alfa has been granted orphan drug designation by the European Commission and the US FDA.
Sanfilippo Syndrome Emerging Therapy Assessment
The dynamics of Sanfilippo syndrome market is anticipated to change in the coming years owing to the improvement in the diagnosis methodologies, raising awareness of the diseases, incremental healthcare spending across the world, and also expects the launch of emerging therapies during the forecast period of 2018–2030
Sanfilippo Syndrome Companies
Companies like Lysogene (LYS-SAF302), Allievex Corporation, Abeona Therapeutics and others are working toward the development of new treatment therapies for Sanfilippo syndrome. While some of the companies have recently shifted their focus toward this indication, yet others are expected to create a significant influence on the market size of during the forecast period.
Sanfilippo Syndrome Report Scope
Table of content
1. Report Introduction
2. Sanfilippo Syndrome
3. Sanfilippo Syndrome Current Treatment Patterns
4. Sanfilippo Syndrome – DelveInsight’s Analytical Perspective
5. Therapeutic Assessment
6. Sanfilippo Syndrome Late Stage Products (Phase-III)
7. Sanfilippo Syndrome Mid Stage Products (Phase-II)
8. Early Stage Products (Phase-I)
9. Pre-clinical Products and Discovery Stage Products
10. Inactive Products
11. Dormant Products
12. Sanfilippo Syndrome Discontinued Products
13. Sanfilippo Syndrome Product Profiles
14. Sanfilippo Syndrome Key Companies
15. Sanfilippo Syndrome Key Products
16. Dormant and Discontinued Products
17. Sanfilippo Syndrome Unmet Needs
18. Sanfilippo Syndrome Future Perspectives
19. Sanfilippo Syndrome Analyst Review
20. Appendix
21. Report Methodology
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