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Sanfilippo Syndrome Pipeline, Emerging therapies and Drugs by DelveInsight

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Sanfilippo Syndrome Pipeline, Emerging therapies and Drugs by DelveInsight

February 03
11:21 2021
Sanfilippo Syndrome Pipeline, Emerging therapies and Drugs by DelveInsight

DelveInsight has launched a new report onSanfilippo 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

  • SLS-005: Seelos Therapeutics
  • LYS-SAF302: Lysogene
  • Tralesinidase alfa (AX-250): Allievex Corporation


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

  • The Sanfilippo Syndrome report provides an overview of therapeutic pipeline activity and therapeutic assessment of the products by development stage, product type, route of administration, molecule type, and MOA type for Sanfilippo Syndrome across the complete product development cycle, including all clinical and nonclinical stages.
  • It comprises of detailed profiles of Sanfilippo Syndrome therapeutic products with key coverage of developmental activities, including technology, collaborations, licensing, mergers and acquisition, funding, designations and other product-related details
  • Detailed Sanfilippo Syndrome research and development progress and trial details, results wherever available, are also included in the pipeline study.
  • Coverage of dormant and discontinued pipeline projects along with the reasons if available across Sanfilippo Syndrome


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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


Related Reports


Media Contact
Company Name: DelveInsight Business Research LLP
Contact Person: Yash Bhardwaj
Email: Send Email
Phone: +919650213330
Address:304 S. Jones Blvd #2432
City: Las Vegas
State: NV
Country: United States

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