Helping Win the Fight for Rare and Orphan Diseases: Amicus Therapeutics

Posted by Cameron on October 2, 2017 in Health |

In a world where many suffer silently from rare diseases, the researchers and scientists at Amicus Therapeutics are raising the bar and creating awareness. This biotechnology company utilizes advanced techniques and offers specialized treatments for a variety of genetic diseases. Finding cures is a 24/7 commitment. This commitment shows in their commitment to:

  • Provide the best care and treatment possible for patients suffering from rare and orphan diseases
  • Support the families of those affected by these diseases
  • Be risk takers and offer choices for those affected by these diseases

Amicus Therapeutics provides advanced product pipelines that state clearly the status and performance rates of each drug tested. At the present time, Amicus Therapeutics is testing three drugs for the treatment of Fabry disease and one drug for treatment of Epidermolysis Bullosa. Drugs and treatment programs go through a seven-phase process. The first phase is the discovery phase where most treatments and drugs are discovered to affect a positive result in patients.


Amicus Therapeutics is now running three programs to uncover cures and treatment for Fabry disease, Epidermolysis Bullosa and Pompe disease (GoogleFinance). Amicus Therapeutics preclinical programs offer researchers and scientists opportunities to test and evaluate the effectiveness of clinical drugs and treatments. Enzyme Replacement Therapy (ERT) has proven to be effective for patients suffering from many rare and orphan diseases.


The scientists and researchers at Amicus Therapeutics work hard at eradicating these rare diseases. Their commitment to researching and targeting the mutated proteins is inspiring. Their platform technologies are specifically designed to stabilize these mutated proteins and alleviate pain.


Listening to the patients and providing much-needed support is a primary focus at Amicus Therapeutics. They understand that the needs of the patient and their families come first. They provide a listening ear and dedicated staff to connect with doctors, hospitals, and other healthcare agencies. They also provide many resources that patients and their families can access to gain valuable information about their disease and treatment options. It is important to the Amicus Therapeutics community to listen to their patients.


Amicus Therapeutics’ patient advocacy has given many patients a voice.

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