Indication name: Phenylketonuria
(PKU)
"Phenylketonuria (PKU) – Market
outlook, Epidemiology, Market Forecast and Competitive Landscape Report – 2020
To 2030 (Markets covered: US, Germany, France, Spain, United Kingdom, Italy,
Japan & China)
Phenylketonuria
(PKU)
is an autosomal recessive inborn error of phenylalanine (Phe) metabolism
resulting from deficiency of phenylalanine hydroxylase (PAH). Mutations in the
PAH gene cause phenylketonuria. The PAH gene provides instructions for making
an enzyme called phenylalanine hydroxylase
Epidemiology- In the United States, PKU
occurs in 1 in 10,000 to 15,000 newborns.
Competitive landscape of Phenylketonuria
(PKU) 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 Phenylketonuria (PKU) 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.
Phenylketonuria (PKU) 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 SYNB1618 Synlogic Phase 2
2 BMN165 BioMarin Pharmaceutical Phase 3
3 RTX-134 Rubius Therapeutics Phase 1
4 CDX
6114 Nestlé Phase 1
5 HMI-102 Homology Medicines, Inc Phase 2
6 CNSA-001 PTC Therapeutics Phase 2"
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