Amicus Therapeutics, Lysosomal Storage Diseases, and How It’s Helping

Fortunately for modern human society, the former threats of many illnesses and ailments that used to threaten peoples’ lives – think smallpox, measles, polio, chicken pox – are long gone, for the most part, thanks to the research and practice of medicine. These ailments were some of the world’s most popular, spurring scientists across the globe to take a collective hand in dealing with them. However, fortunately-unfortunately, some diseases and ailments that are highly problematic don’t affect enough people to make it economically feasible for pharmaceutical giants to develop, research, test, market, and produce treatments or cures for.

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These diseases are called orphan diseases. Several years following the turn of the millennium, Amicus Therapeutics began researching treatments for orphan diseases, mostly those called lysosomal storage diseases, in which microscopic human cells that break down food and other molecules don’t have enzymes that are powerful enough to do the jobs they’re designed to carry out. This results in a host of health problems.

 

Fabry disease is arguably the most popular lysosomal storage disease Amicus Therapeutics has worked on thus far, featuring the painful, quality of life-reducing symptoms of foot and hand pain, hundreds of red spots located on peoples’ torsos, tinnitus, failure to push out ample sweat, lower-quality hearing and eyesight, among other pesky – sometimes downright terrible – symptoms.

 

Fortunately for the few people suffering from Fabry disease, Amicus Therapeutics got migalastat – brand name Galafold – approved for use in Australia just last month (GCReport). This, combined with other approvals and advancements in clinical trials and studies, has spurred high growth in the stock price of Amicus Therapeutics, rising as much as 178% since January 1st, 2017.

 

Amicus Therapeutics was created in tranquil, rural Cranbury, New Jersey, over fifteen years ago in February of 2002 (http://releasefact.com/2017/08/amicus-therapeutics-funds-its-ground-breaking-new-treatment/). In 2007, Amicus Therapeutics “went public,” meaning its stock was available for sale on major stock exchanges, particularly with the NASDAQ trading symbol of FOLD. Amicus Therapeutics currently holds an impressive 900-plus million dollars’ worth of assets, as of its most recent release of financial statements, per June of 2017.