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.

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