Providing Better Treatment for Rare Disease with Amicus Therapeutics

Amicus Therapeutics is one of the largest organizations that deal with the treatment of various genetic diseases. Furthermore, the organization is focused on offering patients with the treatment of unusual diseases. Their main goal is to offer sufficient medical treatment to the rare disease in the society. The organization also accomplished a lot including their first drugs which were approved named Migalastat which treat Fabry disease. In addition, the organization also has set main goals in 2017 such as giving in of J-NDZ for migalastat, establishing clinical arrangement ATB200/AT2221 for Pompe disease and lastly outstandingly finishing the clinical trial which is in phase 3 for Epidermolysis Bullosa.

The organization podium ATB200/AT221 uses an exceptional cure mechanism whereby ATB200 is a practical mixture of alpha-glusosidase enzyme which takes mannose-6 phosphate moieties for uptake addition. Furthermore, AT221 is also chaperone that is cured with ATB200 for stabilizing the component. Amicus Therapeutics is certain that the program will make the organization a top leading rare disease organization by 12% and also approximate a market of $1.2 billion.


Amicus Therapeutics in order to provide better treatment for human genetic diseases they have large professional staff members who create new technology podiums. Mostly the organization aims the mutated proteins when making their medicines. Furthermore, the scientists who work at the organization are well committed to what they do and also it’s their craze hence it’s easy for them to the progress of these programs and they have faith that through using biotechnology and passion to heal rare diseases is important.


Amicus Therapeutics has contributed to the community with the rare disease through partnering with individual patients, health care doctors, caregivers and companies so that they can come up with better strategies to treat rare disease in the society. Also, the organization creates awareness in the community and they also support patients who are suffering from rare disease through helping them find better therapies, offering educative information to them, paying attention to what they say before they start treating them. Lastly, the organizations believe that through taking care of patients and arrange events that will help them they will be able to get better and live healthier.

More about Amicus Therapeutics on

Amicus Therapeutics- Ethical Treatments for Rare Disease

Amicus Therapeutics is a biotechnology company located in Cranbury, New Jersey, who uses a patient centered approach for the advancement of the treatments for the diseases that are deemed rare and devastating. At Amicus Therapeutics they actually live by the motto of “Do Unto Others”, by centering their treatments and patient care in the mindset of individuals or caregivers who have the disease (GoogleFinance). They have developed enzyme replacement treatments for the disease Fabry Disease as well as Epidermolysis Bullosa which they have developed SD-101, as well as treatments for Pompe disease and Lysosomal Storage Disorders.


At Amicus Therapeutics they believe in taking smart risks, working hard, asking questions, supporting patients and their families, and developing the best and most effective treatments for those living with the disease. They believe in taking risk while providing safe treatments and learning from the mistakes they make and reaching further to correct them. In the end, they believe in one thing, Not giving up! and finding a treatment that works in a safe, effective manner.


Amicus Therapeutics executive management team includes their Chief Executive Officer, John F. Crowley who oversees the day to day scientific operations of the company to assure that the focus remains on one person, the patient. He is involved in the hiring, setting the company priorities and vision and well as the execution of the products they develop. Bradley Campbell is the companies Chief Operating Officer and has 15 years experience in creating orphan drugs. Their financial division is headed by Chip Baird who is the Chief Financial Officer. His responsibilities include investment, public relations, project cost and management.


At Amicus Therapeutics they are determined to find cures and treatments for rare and devastating disease while focusing their treatments in a moral, and ethical manner. They provide treatment and trust, in a patient centered environment.