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Fabry Disease – Pipeline Review, H2 2019
Fabry Disease – Pipeline Review, H2 2019, provides an overview of the Fabry Disease (Genetic Disorders) pipeline landscape.
Fabry disease is an X-linked lysosomal disorder that results in abnormal deposits of globotriaosylceramide in blood vessel walls throughout the body. It is caused due to mutations in GLA gene. The GLA gene controls the production of a particular enzyme called alpha-galactosidase A (this enzyme is responsible for breaking down of globotriaosylceramide). Symptoms include skin rash, cramps, gas, diarrhea, heart enlargement, angina, dizziness, headache, nausea, and heat intolerance. Treatment includes enzyme replacement therapy (ERT) and pain management.
Fabry Disease – Pipeline Review, H2 2019, provides comprehensive information on the therapeutics under development for Fabry Disease (Genetic Disorders), complete with analysis by stage of development, drug target, mechanism of action (MoA), route of administration (RoA) and molecule type. The guide covers the descriptive pharmacological action of the therapeutics, its complete research and development history and latest news and press releases.
The Fabry Disease (Genetic Disorders) pipeline guide also reviews of key players involved in therapeutic development for Fabry Disease and features dormant and discontinued projects. The guide covers therapeutics under Development by Companies /Universities /Institutes, the molecules developed by Companies in Pre-Registration, Phase III, Phase II, IND/CTA Filed, Preclinical and Discovery stages are 1, 2, 5, 1, 7 and 2 respectively. Similarly, the Universities portfolio in Unknown stages comprises 1 molecules, respectively.
Fabry Disease (Genetic Disorders) pipeline guide helps in identifying and tracking emerging players in the market and their portfolios, enhances decision making capabilities and helps to create effective counter strategies to gain competitive advantage. The guide is built using data and information sourced from our proprietary databases, company/university websites, clinical trial registries, conferences, SEC filings, investor presentations and featured press releases from company/university sites and industry-specific third party sources. Additionally, various dynamic tracking processes ensure that the most recent developments are captured on a real time basis.
Note: Certain content / sections in the pipeline guide may be removed or altered based on the availability and relevance of data.
– The pipeline guide provides a snapshot of the global therapeutic landscape of Fabry Disease (Genetic Disorders).
– The pipeline guide reviews pipeline therapeutics for Fabry Disease (Genetic Disorders) by companies and universities/research institutes based on information derived from company and industry-specific sources.
– The pipeline guide covers pipeline products based on several stages of development ranging from pre-registration till discovery and undisclosed stages.
– The pipeline guide features descriptive drug profiles for the pipeline products which comprise, product description, descriptive licensing and collaboration details, R&D brief, MoA & other developmental activities.
– The pipeline guide reviews key companies involved in Fabry Disease (Genetic Disorders) therapeutics and enlists all their major and minor projects.
– The pipeline guide evaluates Fabry Disease (Genetic Disorders) therapeutics based on mechanism of action (MoA), drug target, route of administration (RoA) and molecule type.
– The pipeline guide encapsulates all the dormant and discontinued pipeline projects.
– The pipeline guide reviews latest news related to pipeline therapeutics for Fabry Disease (Genetic Disorders)
Reasons to buy
– Procure strategically important competitor information, analysis, and insights to formulate effective R&D strategies.
– Recognize emerging players with potentially strong product portfolio and create effective counter-strategies to gain competitive advantage.
– Find and recognize significant and varied types of therapeutics under development for Fabry Disease (Genetic Disorders).
– Classify potential new clients or partners in the target demographic.
– Develop tactical initiatives by understanding the focus areas of leading companies.
– Plan mergers and acquisitions meritoriously by identifying key players and it’s most promising pipeline therapeutics.
– Formulate corrective measures for pipeline projects by understanding Fabry Disease (Genetic Disorders) pipeline depth and focus of Indication therapeutics.
– Develop and design in-licensing and out-licensing strategies by identifying prospective partners with the most attractive projects to enhance and expand business potential and scope.
– Adjust the therapeutic portfolio by recognizing discontinued projects and understand from the know-how what drove them from pipeline.
4D Molecular Therapeutics Inc
Abeona Therapeutics Inc
Amicus Therapeutics Inc
Chiesi Farmaceutici SpA
Freeline Therapeutics Ltd
Greenovation Biotech GmbH
Idorsia Pharmaceutical Ltd
Moderna Therapeutics Inc
Pharming Group NV
Sangamo Therapeutics Inc
Sigilon Therapeutics Inc
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