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A preview of Amicus Therapeutics

Amicus Therapeutics is a biopharmaceutical company established in 2002 at Cranbury New Jersey. In 2008, Amicus Therapeutics expanded from its single location site in New Jersey to a second research Centre in San Diego. The biotechnology company seeks to treat a range of rare and orphaned disorders. The company product manufacturing systems are anchored on the chaperone advanced replacement therapy (CHART) integrated to enzyme replacement therapies. Amicus Therapeutics enjoys a robust development pipeline program for the treatment of a wide range of human genetic disorders. Additionally, the company had deployed the latest technologies and medicines to cure people with devastating conditions (https://seekingalpha.com/article/4073303-amicus-therapeutics-small-biotech-seems-big-mo). Further, the company envisions to positively impact on the lives of their clients, with the need of patients with rare diseases being the Centre of their incentive science.




Some of the lead products at Amicus Therapeutics include:

  • Migalastat. A lead product targeted to individuals suffering from Fraby disease, based on genetic diagnosis.
  • SD-101. Though in late stage development, the product is set to treat skin blistering and lesions connected with the rare genetic connective tissue disease Epidermolysis Bullosa (EB).
  • ATB200/AT2221 – a paradigm that encompasses enzyme replacement enzymes and pharmacological chaperone to treat Pompe disease.
  • Novel enzyme replacement therapy meant to cure Fabry disease


Ongoing studies:


At Amicus Therapeutics, studies are underway to provide a unique Enzyme Replacement Therapy for Lysosomal Storage Disorders (Crunchbase). Further, a research team is currently working on a Protein replacement therapy for the CDKL5 deficiency. The CDKL5 deficiency is a disorder that manifests itself right from infancy and if not treated may lead to paralysis, visual impairment, scoliosis, sensory impairment and gastrointestinal complications.




Amicus Therapeutics believe in Ethics and compliance in the execution of their daily activities. Further, the company supports programs aimed at fostering patient care with the provision of medical, scientific and educational data to all stakeholders of the company. As part of their Corporate Social Responsibility (CSR), Amicus Therapeutics offers charitable contributions to disadvantaged members of the community and offers educational grants. When undertaking such activities, high priority is given to individuals with or have their beloved ones suffering from CDKL5 deficiency, Epidermolysis Bullosa, and Lysosomal Storage Disorders.


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