GM1 Gangliosidosis – Market outlook, Epidemiology, Competitive Landscape, and Market Forecast Report – 2020 To 2030


 GM1 gangliosidosis is a rare lysosomal storage disorder characterised biochemically by deficient beta-galactosidase activity and clinically by a wide range of variable neurovisceral, ophthalmological and dysmorphic features. Prognosis depends on the type of GM1 gangliosidosis and is extremely poor in the severe infantile form and variable in the chronic adult form of the disease.

·       According to Thelansis, the overall prevalence at birth of GM1 gangliosidosis is estimated to be approximately 1.5 to 2.1 per 100,000 live births.

·       Lack of adequate medical treatment is the unmet need in this space; symptomatic treatment for some neurologic signs and symptoms is available but does not significantly alter the condition’s progression. For example, anticonvulsants may initially control seizures.

·       Few companies like Passage Bio, Axovant Sciences,  etc., are also developing assets in the pre-clinical stages.

 

The competitive landscape of GM1 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 GM1 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.

GM1 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: GM1 Gangliosidosis – Market outlook, Epidemiology, Market Forecast, and Competitive Landscape Report – 2020 To 2030

S. No    Asset               Company                                 Stage

1          ORMD-0801     Oramed, Ltd.     Phase 2

2          EDP-305           Enanta Pharmaceuticals            Phase 1

3          HepaStem        Promethera Biosciences            Phase 2

4          TERN-201         Terns Pharmaceuticals, Inc.       Phase 2

5          Namodenoson   Can-Fite BioPharma      Phase 2

6          BIO89-100        89bio, Inc.         Phase 2

7          VK2809 Viking Therapeutics, Inc.           Phase 2

8          AZD2693          AstraZeneca     Phase 1

9          BMS-986263     Bristol-Myers Squibb      Phase 2

10        BOS-580          Boston Pharmaceuticals Phase 2

Continued.

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