Amicus Therapeutics Mixes Research With Humanity

Amicus Therapeutics, Inc. is an international biotechnology company whose primary goal is to treat disastrous rare and orphan diseases, and Migalastat is its breakout drug (YahooFinance). Amicus Therapeutics’ drug therapy research targets mutated proteins in their search to develop new medicines.


Proteins are needed to maintain life. Hair, nails are made of proteins. Proteins build bones, muscles, skin, blood, cartilage, and enzymes. Lysosomes are sacs inside cells. They contain enzymes that accelerate chemical reactions which break down (metabolize) excess sugar and fats (lipids), substances which are the cell’s food.


The cells are basic units of life, need metabolizing substances to maintain cell life. There are specialized cells that form tissues, which form the organs and make up the body. When the life of cells is compromised, so are the organs and the tissues of the body, as the result of the damage to the cells’ metabolic process.


There is a rare cell disorder named Fabry disease which is a genetic Lysosomal storage disease. There are fifty lysosomal storage diseases. Amicus Therapeutics has concentrated on Fabry disease.


If this disease is not adequately treated, the result will be damage to tissues, and organs are causing harm to the body. The symptoms of Fabry disease include disruption in intellectual and physical development, deformities of the face and bones, stiffness and pain in joints, problems with breathing, disruptions in vision and hearing and seizures.


Amicus Therapeutics has developed a drug that fights this rare genetic disease. Recently, the FDA is permitting Amicus Therapeutics to submit a new drug application for Migalastat, an oral precision medicine, to treat Fabry disease. Migalastat works as an enzyme replacement therapy.


Amicus Therapeutics is on a constant quest to find the ultimate cure or treatment in cooperation with practitioners, patients, caregivers and the rare disease community organizations. In this last respect, Amicus Therapeutics has formed an Amicus Patient and Professional Advocacy platform (,-NJ-jobs.html).


According to Amicus Therapeutics’ Chairman and CEO John F. Crowley, Amicus Therapeutics takes its research seriously in having a personal relationship with those who suffer from lysosomal storage diseases, placing pictures of their patients in their offices around the world.

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