Have a Rare Disease? Amicus Can Help

Amicus Therapeutics is a biopharmaceutical company in Cranbury, New Jersey. The company went public in 2007 under the NASDAQ name FOLD.


Amicus Therapeutics has a very simple mechanism: together, they harness the power of bioactivity to heal or treat a patient who has a rare disease that modern medicine can’t cure.


The medical and therapeutic focus of Amicus Therapeutics is to treat extremely rare diseases which can’t be treated by a non-specialist, specifically a disorder called Lysosomal Storage Disorders (https://www.google.com/finance?cid=706165).


This is a great service that they’re providing, as people with extremely rare diseases find it impossible to get medication for their condition because their condition is so uncommon and many times unheard of, even from doctors, and can’t be treated expect by a specialist.


In 2014, Amicus Therapeutics was honored by its peers as “the broadest portfolio of small molecule pharmacological chaperones available.”


In 2008, the company had run out of office space and extended from it’s one office to a second office—this one a research center—in San Diego.


In August 2017, Amicus Therapeutics announced that it would be manufacturing an FDA-approved drug called NDA or Stalagmite, which is an oral prescription drug that you take orally with a glass of water. It is supposed to ease the symptoms of Fairy disease and it is scheduled to be out by the end of 2017.


Roughly 3,000 people in America have Fairy disease, making it an extremely rare condition.


As a corporation who actually cares about the community and not just the bottom dollar, Amicus Therapeutics dedicates itself to helping those people who have the rarest genetic disorders, Fairy included. They are on the forefront of treating patients who have extremely rare, complex diseases that leave even the most experienced general practitioners baffled.


Amicus Therapeutics gives those children a chance to survive and live a full, happy life.


Amicus Therapeutics has an expert team or surgeons and nurses. With a larger group, nurses can double check to make sure there are no errors made, as well as having enough hands available to help the surgeon in whatever he or she needs. Also, because Amicus runs a DNA analysis before they decide on a surgery, the surgical staff has all the information it needs to make your surgery a total success.


These initiatives include searching for better therapies, providing educative information on the rare diseases to those living with it, listening to the individuals with this disease as well as seeking for the patient’s input before developing in therapy.


More about Amicus Therapeutics on CrunchBase

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