Amicus Therapeutics: The Fight Against Orphan Diseases

When it comes to dealing with rare diseases, Amicus Therapeutics is consistently front and center. The company is currently in the process of developing various technologies to treat aspects of these diseases. Many of these advancements are designed to make life easier for people living with Fabry disease or conditions such as Epidermolysis Bullosa.

What makes Amicus Therapeutics stand out in the industry is their clear dedication to the needs of the people within the rare disease community; everything the company does is to promote better living for both the patients and those that care for them.


Amicus Therapeutics builds its organization and portfolio of clinical programs around science that can mitigate the worst aspects of various rare diseases.


Experts have noted that at this time, the company likely has the broadest portfolio of chaperones for small molecules in the whole industry. The focus of Amicus Therapeutics is primarily developing revolutionary enzyme replacement therapies. Such advancements can help stabilize adverse conditions Fabry disease and the like.


Over time, Amicus has expanded to more research sites including San Diego and acquired competitors and other companies with similar interests and goals. This has allowed Amicus Therapeutics to enhance its available research options and the intellectual properties that were available to advance other research.


In addition to its own capital, Amicus has been awarded several grants and other monies thanks to its successes and cutting-edge research. Various foundations dedicated to neurological research and other fields provided these additional finances knowing that Amicus would be most likely to make a breakthrough.


Amicus Therapeutics doesn’t simply do solid research, they’re also well-known for their dedication to patient advocacy. The company provides initiatives that work to empower patients living with rare diseases. Along with their research, Amicus provides resources and support services to deal with not only the physical aspects of the diseases but the emotional toll they can take as well.


The company takes a comprehensive approach to this by listening to the stories of those affected and their families and incorporating that patient input into their therapies (Crunchbase). Amicus Therapeutics is currently developing several treatment options hoping to provide massive benefits to those dealing with rare and orphan diseases today.

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Categories: Medication