Amicus Therapeutics – We Believe

While an ongoing struggle with cancer, HIV/AIDS, diabetes, and many other common diseases rages on, many overlook those suffering from lesser-known genetic conditions ( Amicus Therapeutics is on the forefront of the battle against these orphan diseases. Based out of New Jersey, this company focuses its development on Chaperone-Advanced Replacement Therapy (CHART™)and enzyme replacement therapies (ERTs), essentially fighting the disorders on a molecular level.


There are many other companies trying to combat these illnesses, but Amicus Therapeutics is addressing the unmet needs that come with each disease. Focusing on Fabry disease, Pompe disease, and other Lysosomal Storage Disorders (LSDs), they have developed several treatments. Among these treatments are ATB200/AT2221, an enzyme replacement therapy for Pompe disease, and SD – 101, a proprietary topical formulation for the painful skin blistering and lesions that come with Epidermolysis Bullosa (EB). Both of these studies are still in the clinical trial phases, however, there is one treatment that is nearing commercial distribution. Migalastat, also known as Galafold, treats Fabry disease, an x-linked genetic disorder that progressively causes irreversible organ damage. Migalastat is an oral pharmacological chaperone that in a double-blind, placebo-controlled study, was proven to reduce the substrate GL-3 (the cause of cell damage with Fabry disease)by ≥50%. This is just a small example of the great strides Amicus Therapeutics is making towards orphan disease therapies.


Along with supporting the disease communities, Amicus Therapeutics also supports the individuals and families of those afflicted with these illnesses. On their website you can read about the struggles and pains some patients suffer through, but also the hope that comes with taking small steps to better treatment in the future. Looking at the big picture, it’s clear to see that Amicus Therapeutics is not just a biopharmaceutical company (Twitter). They are a family that fights for those who suffer from diseases that most of us know nothing about. With a small point from their mission statement, “We believe. . . in the fight, in our future, in each other,” they inspire and work today, for a better tomorrow.

More at

Leave a Reply