Posts in Category: Diseases

Curing Rare Diseases with Amicus Therapeutics

Amicus Therapeutics, Inc. is a leading new frontier global biotechnology company based in Cranbury, New Jersey. This innovative company researches new discovery findings, the development, and adopting therapies for devastating rare and orphan diseases. Amicus Therapeutics has a vigorous line of one-of-a-kind small molecules called ‘pharmacological chaperones.’ The chaperones provide dual-treatment to bind, stabilize and increase the activity of a patient’s own deformed enzymes to potentially improve enzyme replacement therapy outcome.

 

Amicus Therapeutics, Inc., research and development processes began as a result of Megan Crowley, who was diagnosed with Pompe disease which she had been suffering with since the age of one. Pompe is a progressive muscle and respiratory weakening disease with a high mortality rate. Megan’s father, John Crowley is now the CEO of this biotechnology company which has a pipeline of therapies for human genetic diseases like his daughter has. Mr. Crowley is determined to find a cure for his daughter and many others who suffer from rare, unexplored diseases (https://www.glassdoor.com/Reviews/Amicus-Therapeutics-Reviews-E26068.htm). His quest has made Amicus Therapeutics a leader for the drug industry and has made them champions in the development of new medical treatments and programs.

 

Fabry is also an early age disease that affects millions around the world, just like the Pompe disease. Fabry disease leads to progressive, irreversible organ damage, typically involving the nervous, cardiac, and renal systems, as well as multiple other tissues. Both diseases require daily medical assistance to manage and control the complications of these devastating diseases affecting multiple organ systems. To find a cure for the Fabry disease, the Pompe disease, and other chronic diseases, Amicus Therapeutics is investigating enzyme replacement therapy, influencing chaperone advanced replacement therapy, taking a lead in the clinical development of topical medicine to treat genetic connective tissue disorders, including investigating many other scientific first treatments and discovery programs.

 

Amicus Therapeutics is leading the fight in developing a topical therapy for the treatment of lesions and blisters that appear on the skin and in some cases on the lining of other body organs. This rare, chronic disease is known as Epidermolysis Bullosa. Epidermolysis Bullosa can potentially become disfiguring, and in some cases fatal. Amicus Therapeutics has also taken the lead recently in promising finds for the underlying cause of muscle damage and weakness in Pompe patients. This pioneering company has a three-fold promise, which is to remain at the forefront of therapies for rare and orphan diseases, to build long-term value for stakeholders and to foster teamwork and respect for each individual’s contribution.

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