Indication name: Angelman
syndrome (AS)
Angelman syndrome (AS) – Market
outlook, Epidemiology, Market Forecast and Competitive Landscape Report – 2019
To 2030 (Markets covered: US, Germany, France, Spain, United Kingdom, Italy,
Japan & China)
Angelman syndrome (AS) is a rare
neuro-genetic disorder, Thelansis estimated that disease occurs in one in
15,000 live births or 500,000 people worldwide. Angelman syndrome is estimated
to be out of total US population, people affected by Angelman syndrome are
28,404.
In most cases of Angelman
syndrome, these genetic changes appear to occur randomly (sporadically) but in
about 3-5% they can be inherited. In approximately 70-75 percent of cases there
is a microdeletion of region 15q11-13 of the maternally-derived chromosome 15
that includes deletion of the UBE3A gene.
In about 1 percent of cases, a
deletion of this chromosomal region may occur due to a complex chromosomal
rearrangement, in which a segment of chromosome 15 breaks off and moves to
another chromosomal location. Approximately 2-5 percent of Angelman syndrome
cases are caused by uniparental disomy, an abnormality in which a person
receives both copies of a chromosome from one parent instead of receiving one
from each parent.
KOLs
insights of Angelman syndrome (AS) 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.
Competitive landscape of Angelman
syndrome (AS) 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.
Angelman syndrome (AS) Market
Forecasting: 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 GTX-102 GeneTX Biotherapeutics,
LLC Phase 2
2 RO7248824 Hoffmann-La Roche Phase 1
3 Gaboxadol Ovid Therapeutics Inc. Phase 3
4 Circadin Neurim Pharmaceuticals Ltd. Phase
3
5 NNZ-2591 Neuren Pharmaceuticals Ltd Phase 1
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