Amicus Therapeutics: Fighting Against Rare Diseases

Amicus Therapeutics is working diligently toward the treatment and cure of rare and orphan diseases. Using the latest technology and medicine available in the field of biotechnology, rare diseases such as Pompe disease and Epidermolysis Bullosa are being treated. Amicus Therapeutics focuses its research and treatment on a collection of disorders known as lysosomal storage disorders. Since the launch of the breakthrough medication Galafold in 2014, Amicus Therapeutics continues to deliver successful medication developments. Follow Amicus Therapeutics on

The biopharmaceutical company received grants from multiple foundations to aid in the fight against rare disease. Such foundations as The Michael J. Fox Foundation and the Alzheimer’s Drug Discovery Foundation funded support for research and treatment efforts to Amicus Therapeutics. Scientists at Amicus Therapeutics combine knowledge and passion for treating rare genetic diseases. Amicus Therapeutics develops and personalizes medicine to treat individuals according to their specific genetic disease. Know more on about Amicus Therapeutics.

To treat lysosomal storage disorders, scientists at Amicus Therapeutics are developing an Enzyme Replacement Therapy. While Enzyme Replacement Therapy is being used in treatment programs for Fabry and Pompe disease, Amicus Therapeutics continues to reach new advancements in biotechnology. In preclinical studies a protein replacement therapy is being developed to aid in treating genetic mutations in children. Many pills and vaccines are in the late-stages of development for multiple rare diseases. Amicus Therapeutics is also in the late-stage development of SD-101 Cream for the treatment of Epidermolysis Bullosa. SD-101 Cream is the first drug to receive Breakthrough Therapy and orphan drug designation from the U.S. Food and Drug Administration.


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