GM2 Gangliosidosis – Market outlook, Epidemiology, Competitive Landscape and Market Forecast Report – 2020 To 2030

GM2 Gangliosidosis – Market outlook, Epidemiology, Competitive Landscape and Market Forecast Report – 2020 To 2030

Description:

Indication name: GM2 Gangliosidosis

GM2 gangliosidosis is an autosomal recessive metabolic disorder due to β-hexosaminidase deficiency. GM2 gangliosidosis is divided into three clinical subtypes according to the age at onset. In general, the later the disease occurs, the more slowly it progresses. Type 1 (infantile type) begins in the first year of life with rapidly progressive diffuse neurological deterioration and death before 4 years of age. GM2 gangliosidosis is caused by deficiency of (beta-1,4 N-acetyl galactosaminidase) hexosaminidase activity. The two isoenzymes are called hexosaminidase A and B. Typical infantile forms of GM2 gangliosidosis are Tay–Sachs disease and Sandhoff disease.

·       The incidence of the Tay Sachs variant in the general population is estimated to be 1.75 cases per 250,000 live births, however, Sandhoff-variant incidence has been reported at 1 to 2 cases per 350,000 patients.

·       The most common initial symptoms were developmental arrest (83%), abnormal startle response (65%), and low muscle tone (60%).

The competitive landscape of GM2 Gangliosidosis includes country-specific approved and pipeline therapies. Any asset/product-specific designation or review and Accelerated Approval are tracked and supplemented with analyst commentary.

KOLs insights of GM2 Gangliosidosis across 8 MM market from the centre of Excellence/ Public/ Private hospitals participated in the study. Insights around current treatment landscape, epidemiology, clinical characteristics, future treatment paradigm, and Unmet needs.

GM2 Gangliosidosis 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: GM2 Gangliosidosis – Market outlook, Epidemiology, Market Forecast and Competitive Landscape Report – 2020 To 2030

S. No    Asset               Company                                 Stage

1          TSHA-101         Taysha Gene Therapies, Inc.     Phase 2

2          venglustat GZ402671    Genzyme, a Sanofi Company     Phase 3

3          miglustat           Actelion Phase 2

4          AXO-AAV-GM2 Sio Gene Therapies       Phase 2

 

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