Fabry Disease is a rare genetic disorder that prevents the body from making an enzyme called alpha-galactosidase A. This enzyme is responsible for breaking down a type of fat called globotriaosylceramide (Gb3 or GL-3) into building blocks that the body’s cells can use. Fabry Disease can have many symptoms because it affects cells throughout the body. The symptoms include: Chronic pain — usually a burning or tingling sensation — in the hands and feet, Clusters of small, dark red spots in various locations on the skin, Opaque corneas, resulting in cloudy-looking eyes and problems with eyesight, Ringing in the ears, and hearing loss. Fabry Disease is caused by a mutation of the GLA gene, which encodes for the alpha-galactosidase A enzyme. The mutation usually makes the enzyme unable to function, although in mild cases, it may function to some degree. The disease is inherited in an X-linked dominant manner, which means that the gene involved is situated on the X chromosome.
DelveInsight’s, “Fabry Disease Pipeline Insight, 2021,” report provides comprehensive insights about 15+ companies and 15+ pipeline drugs in Fabry Disease pipeline landscape. It covers the pipeline drug profiles, including clinical and nonclinical stage products. It also covers the therapeutics assessment by product type, stage, route of administration, and molecule type. It further highlights the inactive pipeline products in this space.
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Some of Fabry Disease Companies are:
DelveInsight’s Fabry Disease report covers around 15+ products under different phases of clinical development like:
Some of Fabry Disease Drugs are:
Fabry Disease Report Key Questions:
Table of Contents:
Introduction
Executive Summary
Fabry Disease: Overview
Pipeline Therapeutics
Therapeutic Assessment
Fabry Disease – DelveInsight’s Analytical Perspective
In-depth Commercial Assessment
Fabry Disease Collaboration Deals
Late Stage Products (Preregistration)
Pegunigalsidase alfa: Protalix Biotherapeutics
Drug profiles in the detailed report…..
Mid Stage Products (Phase II)
Venglustat: Sanofi
Early Stage Products (Phase I/II)
4D 310: 4D Molecular Therapeutics
Inactive Products
Fabry Disease Key Companies
Fabry Disease Key Products
Fabry Disease- Unmet Needs
Fabry Disease- Market Drivers and Barriers
Fabry Disease- Future Perspectives and Conclusion
Fabry Disease Analyst Views
Appendix
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