Amicus Therapeutics: Passionate, Patient-Centered, Personalized, and Innovative Care for Orphan Diseases

Amicus Therapeutics, a world-renowned biotechnology company, specializes in advancing care and therapies for uncommon diseases. The company channels its investments in research and development of treatments for a variety of genetic diseases.


Amicus Therapeutics launched the inaugural EMA approved precision medicine, Galafold, that treats Fabry disease (MarketWatch). It partnered with the European Medicines Agency and the FDA. The New England Journal of Medicine contains all the data used in the trial of Galafold. The company is expecting FDA approval for the drug.


Presently, Amicus Therapeutics is working on completing a treatment plan for Pompe’s disease and trials readouts for epidermolysis bullosa before the end of 2017. SD-101 is the market therapy that is being developed to conquer Epidermolysis Bullosa, a connective tissue disorder. Amicus Therapeutics is using genetically-engineered proteins and their Chaperone-Advanced Replacement Therapy systems to create enzyme replacement therapy drugs for Pompe, Lysosomal Storage Disorders, and Fabry disease.


The programs set up by Amicus Therapeutics have three characteristics. They treat orphan diseases, employ first class technology, and the results from the clinical data show meaningful progress for patients.


Personal and direct relations are at the core of the organizational culture. The team at the company provides compassionate and customized care for patients and their support systems. Each of the global offices has pictures and narratives that tell the patients’ lives and struggle with specific disorders.


Amicus Therapeutics incorporate a patient-focused approach in decision making ( For every decision, the management tries to get a patient’s perspective. The leadership explores the risks, benefits and other impacts before making the decisions.


The team at Amicus Therapeutics comply with the ethics standards. They are persistently looking for ways to provide better care and innovate. The passion for helping the needy unites the employees at the company. They draw their inspiration from helping people with rare diseases improve their conditions. Invention and healing motivate the staff while the dedicated leadership drive developments and aspirations to scale new heights.


Amicus Therapeutics passionately embraces innovations, takes risks, pushes its ideas, creates value for all and uses alternative approaches. The company is not afraid of making its technology redundant. Mistakes act as building blocks for future success.