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Fabry Disease - Pipeline Review, H1 2018

Press Release   •   Apr 10, 2018 13:45 IST

Global Markets Directs latest Pharmaceutical and Healthcare disease pipeline guide Fabry Disease Pipeline Review, H1 2018, provides an overview of the Fabry Disease Genetic Disorders pipeline landscape.

Fabry disease is an Xlinked 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 alphagalactosidase A this enzyme is responsible for breaking down of globotriaosylceramide.

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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.

Report Highlights

Global Markets Directs Pharmaceutical and Healthcare latest pipeline guide Fabry Disease Pipeline Review, H1 2018, 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 PreRegistration, Phase III, Phase II, Phase I and Preclinical stages are 2, 1, 4, 1 and 5 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 Global Markets Direct’s proprietary databases, company/university websites, clinical trial registries, conferences, SEC filings, investor presentations and featured press releases from company/university sites and industryspecific third party sources. Additionally, various dynamic tracking processes ensure that the most recent developments are captured on a real time basis.


  • 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 industryspecific sources.
  • The pipeline guide covers pipeline products based on several stages of development ranging from preregistration 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

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Table of content

List of Tables
List of Figures
Global Markets Direct Report Coverage
Fabry Disease Overview
Fabry Disease Therapeutics Development
Pipeline Overview
Pipeline by Companies
Pipeline by Universities/Institutes
Products under Development by Companies
Products under Development by Universities/Institutes
Fabry Disease Therapeutics Assessment
Assessment by Target
Assessment by Mechanism of Action
Assessment by Route of Administration
Assessment by Molecule Type
Fabry Disease Companies Involved in Therapeutics Development
Amicus Therapeutics Inc
Genzyme Corp
greenovation Biotech GmbH
iBio Inc
Idorsia Ltd
JCR Pharmaceuticals Co Ltd
Pharming Group NV
Protalix BioTherapeutics Inc
Resverlogix Corp
Sangamo Therapeutics Inc
Fabry Disease Drug Profiles
agalsidase alfa Drug Profile
Product Description
Mechanism Of Action

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