Menkes disease (MD) – Market outlook, Epidemiology, Competitive Landscape and Market Forecast Report – 2020 To 2030
Menkes disease (MD) is a lethal multisystemic disorder of copper metabolism. Progressive neurodegeneration and connective tissue disturbances, together with the peculiar ‘kinky’ hair are the main manifestations. MD is inherited as an X-linked recessive trait, and as expected the vast majority of patients are males. MD occurs due to mutations in the ATP7A gene and the vast majority of ATP7A mutations are intragenic mutations or partial gene deletions. ATP7A is an energy-dependent transmembrane protein, which is involved in the delivery of copper to the secreted copper enzymes and in the export of surplus copper from cells. Severely affected MD patients die usually before the third year of life. A cure for the disease does not exist, but very early copper-histidine treatment may correct some of the neurological symptoms.
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Menkes disease is a rare condition with
incidence estimates ranging from 0.75 to 1.27 cases per 100,000 live births in
the USA. It is an inborn disorder of copper metabolism with multisystem
ramifications.
The competitive
landscape of Menkes disease (MD) 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 Menkes
disease (MD) 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.
Menkes
disease (MD) 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 CUTX-101, copper histidinate Cyprium Therapeutics, Inc. Phase
3
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