Amicus Therapeutics, Inc. (NASDAQ: FOLD) is an American biopharmaceutical company that deals with the treatment of lysosomal storage disorders. The company concentrates on enzyme replacement therapies, which they use in treating a wide range of rare and orphan diseases. The company uses chaperone-advanced replacement therapy in their developments too.
Amicus Therapeutics Products
Amicus Therapeutics applies technology for the treatment of human genetic diseases and stabilization of mutated proteins. One of their most successful products is the Migalastat, which is a personalized drug for treating Fabry disease. The medicine, which is in its final development stage, treats based on Fabry’s genetic diagnosis.
The company has also developed SD-101, which is also in its final stage of development. The SD-101 is for treating Epidermolysis Bullosa, which it is the first therapy, of its kind, for treating the rare genetic cognitive disorder (WeeklyOpinion).
How Amicus Therapeutics Uses Technology to Treat Lysosomal Storage Disorder (LSD)
LSD occurs when certain lysosomal enzymes mutate causing the proteins in the lysosomal channel to degrade into smaller pieces that can form new molecules. When this happens, the proteins of the patient become unstable, which result in a number of complications including cell death, substrate accumulation, and cellular function disruption.
To treat the condition, Amicus Therapeutics has developed pharmacological chaperone technology, which selectively binds itself to a target enzyme and increases its stability (https://finance.yahoo.com/quote/FOLD?ltr=1). Other than holding enzymes, pharmacological chaperones are also used to transport endogenous and exogenous enzymes in cell locations they are required.
The Company’s Base of Success
Being in the forefront of providing a cure for rare and orphan diseases has made Amicus Therapeutic operate on a strong foundation that helps achieve their strong vision. The company is made up of a team of professionals who are led by John Crowley as the chairman and CEO.
The company ensures that every program they try must meet three characteristics. It must focus on rare and devastating disorders, it must be the best technology in its class, and it must prove useful in treating the designated condition. In addition, Amicus Therapeutics creates a close connection with their patients, families, and caregivers.