Indication name: Adult
polyglucosan body disease (APBD)
"Adult polyglucosan body disease (APBD)
– Market outlook, Epidemiology, Market Forecast and Competitive Landscape
Report – 2020 To 2030 (Markets covered: US, Germany, France, Spain, United
Kingdom, Italy, Japan & China).
Adult
polyglucosan body disease (APBD) is a rare, genetic disorder characterized
by a deficiency of glycogen-branching enzyme, resulting in the accumulation of
polyglucosan bodies in muscle, nerve and various other tissues of the body.
Polyglucosan bodies are composed of large, complex, sugar-based molecules.
Mutations in the GBE1 gene cause APBD. The GBE1 gene provides instructions for
making the glycogen branching enzyme. This enzyme is involved in the production
of a complex sugar called glycogen, which is a major source of stored energy in
the body. The two most common mutations are p.Y329S and the deep intronic
mutation.
Epidemiology- Approximately 200 affected
individuals have been diagnosed worldwide.
Competitive landscape of Adult polyglucosan
body disease (APBD) includes country specific approved as well as pipeline
therapies. Any asset/ product specific designation or review and Accelerated
Approval are being tracked and supplemented with analyst commentary.
KOLs insights of Adult polyglucosan body
disease (APBD) across 8 MM market from center of Excellence/ Public/ Private
hospitals participated in the study. Insights around current treatment
landscape, epidemiology, clinical characteristics, future treatment paradigm
and Unmet needs.
Adult polyglucosan body disease (APBD)
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 Triheptanoin Ultragenyx Pharmaceutical Inc Phase 2
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