Krabbe Disease – Market outlook, Epidemiology, Competitive Landscape and Market Forecast Report – 2020 To 2030


 Krabbe disease is a lysosomal disorder that affects the white matter of the central and peripheral nervous systems. It includes infantile, late-infantile/juvenile, and adult forms.

Etiology-

Krabbe disease is caused by changes (mutations) in the GALC gene. This gene provides instructions for making an enzyme called galactosylceramidase, which breaks down a certain type of fat that is primarily found in the brain and kidneys. Mutations in the GALC gene lead to reduced levels of the galactosylceramidase enzyme which can cause a build-up of toxic fats in the cells of the brain and other tissues.

Rarely, infantile Krabbe disease is caused by a mutation in the prosaposin (PSAP) gene (10q21-q22), encoding sphingolipid activator protein saposin-A, necessary for GALC activity.

Epidemiology-

It has an estimated prevalence of 1/100,000 in the Northern European population (higher in certain populations) and a worldwide incidence of 1/100,000-1/250,000 live births. The infantile form is the most common form and accounts for 85-90% of cases in the Northern European population.

 

The competitive landscape of Krabbe Disease 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 Krabbe Disease across 8 MM market from the center of Excellence/ Public/ Private hospitals participated in the study. Insights around current treatment landscape, epidemiology, clinical characteristics, future treatment paradigm, and Unmet needs.

Krabbe Disease 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: Krabbe Disease – Market outlook, Epidemiology, Market Forecast, and Competitive Landscape Report – 2020 To 2030

S. No    Asset               Company                                 Stage

1          FBX-101           Forge Biologics, Inc       Phase 1/2

2          PBKR03            Passage Bio, Inc.          Phase 1/2

3          MGTA-456        Magenta Therapeutics, Inc.        Phase 2

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