Amicus Therapeutics recently has its prescription drug approved called Galafold by Health Canada. This oral drug is used to treat a rare disease called Fabry disease. It is a disease that causes a defect in an enzyme in the body. This leads to an excess amount of fat in the body called Gb3 and G-L3. The drug will be available to Canadian patients (https://seekingalpha.com/article/4073303-amicus-therapeutics-small-biotech-seems-big-mo).
This is a positive step for Canadians suffering from this disease said John F. Crowley, Chairman, and Chief Executive Officer. The company looks forward to making the medication accessible to other countries. The drug works by stabilizing the enzyme so that it clears the body of the fat buildup.
The drug was approved after several clinical trials. Dr. Daniel Bichet from the University of Montreal said that after years of treating a patient with Fabry disease he was glad to see the drug approved. There are many people that will benefit from treatment from this drug.
John Crowley, CEO of the company became a biotech entrepreneur to help his two children with a rare disease called Pompe disease. It is a neuro-muscular disease that can be fatal. He became involved in the industry when both children were diagnosed with the disorder. At the time there was no treatment or cures.
Their research leads to a treatment that both children have been on for a decade and lead to saving their lives. The company collaborates with organizations, doctors, patients, and caregivers. They listen and learn about the rare diseases from patients and caregivers. They do extensive research and always look for new and better therapies (Indeed). The company often use patients in clinical studies and help patients manage their daily activities.
The company staff is very passionate about the work they do. The will continue to come up with new innovative treatments for rare diseases in the future.