Sanfilippo syndrome – Market outlook, Epidemiology, Market Forecast and Competitive Landscape Report – 2019 To 2030

 

Indication name: Sanfilippo syndrome

Sanfilippo syndrome – Market outlook, Epidemiology, Market Forecast and Competitive Landscape Report – 2019 To 2030

Sanfilippo syndrome is a rare, inherited disorder also known as mucopolysaccharidosis type III (MPS III), is classified as a lysosomal storage disorder (LSD). In these disorders, genetic variations disrupt the normal activity of lysosomes in human cells. Mutations in the GNS, HGSNAT, NAGLU, and SGSH genes cause MPS III. These genes provide instructions for making enzymes involved in the breakdown of large sugar molecules called glycosaminoglycans (GAGs, formerly called mucopolysaccharides).

Thelansis Epidemiology study indicates, MPS III is the most common form of mucopolysaccharidosis; the estimated incidence of all four types combined is 1 in 70,000 newborns.

Competitive landscape of Sanfilippo syndrome includes country specific approved as well as pipeline therapies. Any asset/ product specific designation or review and Accelerated Approval are being tracked and supplemented with analyst commentary.

KOL insights of Sanfilippo syndrome across 8 MM market from center of Excellence/ Public/ Private hospitals participated in the study. Insights around current treatment landscape, epidemiology, clinical characteristics, future treatment paradigm and Unmet needs.

Sanfilippo syndrome Market Forecast: Patient Based Forecast Model (MS. Excel Based Automated Dashboard) which Data Inputs with sourcing, Market Event and Product Event, Country specific Forecast Model, Market uptake and patient share uptake, Attribute Analysis, Analog Analysis, Disease burden and pricing scenario, Summary and Insights.

Read more: Sanfilippo syndrome – Market outlook, Epidemiology, Market Forecast and Competitive Landscape Report – 2019 To 2030

 

S. No        Asset        Company        Partner        Phase

1                 ABO-101        Abeona Therapeutics, Inc.        N/A        I/II

2                 Tralesinidase Alfa        BioMarin Pharmaceutical Inc.        N/A        I/II

3                 AMT-110        uniQure N.V.        N/A        Development Outside U.S.

4                 EGT-201        Esteve, S.A.        N/A        Preclinical

5                MPS IIIB AAV Gene Therapy (Amicus/Penn)        Amicus Therapeutics, Inc.        UPenn        Preclinical

6                OTL-202        Orchard Therapeutics Limited        N/A        Preclinical