Metachromatic Leukodystrophy – Market outlook, Epidemiology, Competitive Landscape and Market Forecast Report – 2020 To 2030
A rare lysosomal disease characterized by accumulation of sulfatides in the central and peripheral nervous system due to deficiency of the enzyme arylsulfatase A, leading to demyelination.
Etiology-
This
condition is inherited in an autosomal recessive pattern and is caused by
mutations in the ARSA and PSAP genes.
Epidemiology-
The
true prevalence rate of MLD is unknown but is estimated to be between 1 in
40,000 and 1 in 160,000. The Navajo also have a higher prevalence rate of 1 in
every 2,500 people. In certain populations in the Middle East, these numbers
may be even higher.
The competitive
landscape of Metachromatic Leukodystrophy 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 Metachromatic
Leukodystrophy 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.
Metachromatic
Leukodystrophy 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.
S. No Asset Company Stage
1 rhASA Shire Phase 1
2 OTL-200 Orchard
Therapeutics Phase 1/2
3 hematopoietic stem cell infusion Talaris Therapeutics Inc. Phase
1/2
4 MGTA-456 Magenta
Therapeutics, Inc. Phase 2
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