In the forefront of advanced therapy, Amicus Therapeutics Inc is leading the way as a biotechnology company. They help to treat a range of rare and orphaned diseases which people are forced to deal with yearly due to genetic diseases.
Currently their number one candidate is Migalastat, the late stage development medicine that is being used in individuals who have Fabry disease. SD-101 is a product that is a candidate with late stage development that is hoped to be the first in class to market therapy in the rare connective tissue disorder that is passed down genetically from one person to another (https://www.crunchbase.com/organization/amicus-therapeutics).
Amicus Therapeutics will work with to ensure that individual patients and their caregivers are doing and receiving everything they can in order to support individuals who have rare diseases. They will work to ensure that families, patients, caregivers and others have the access they need in order to have support and services across all experiences with their disease. It is the goal of Amicus Therapeutics to deliver healing even further than the disease.
Amicus Therapeutics is also looking for the better and new therapies that are out there for patients suffering from genetic disorders and diseases. They will incorporate the input from their patients during clinical trials of therapies which offer research advances in the drug developments processes. They will also provide any useful information as well as resources to help to manage what each patient is met with daily when it comes to living with genetic diseases year after year.
Amicus Therapeutics has a number of tactics that they use to deliver the benefits that their patients and their families gets from them (YahooFinance). There are several treatment options being developed at one time to help ensure that rare diseases are being addressed each year. Amicus also searches for ways that they can be active within the community by participating in events which educate and support patients who might not know that they are out there. They push to advocate for patients and their families as they start to navigate within the rare disease community. If you are faced with or know someone with a rare genetic disorder, Amicus Therapeutics could be what you need.