Amicus Therapeutics’ mission is to transform the lives of patients with rare and orphan diseases

Amicus Therapeutics’ mission is to transform the lives of patients with rare and orphan diseases by developing innovative, high quality therapies. The company envisions a future where advanced science and compassionate care come together to provide effective treatments for all patients in need.Amicus Therapeutics focuses on several key therapeutic areas, utilizing advanced technologies and platforms to develop novel treatments:Amicus has developed Galafold® (migalastat), the first oral precision medicine for Fabry disease. Galafold® is designed to treat patients with amenable mutations, providing a new standard of care for this rare genetic disorder.The company is advancing AT−GAA, a next−generation therapy for Pompe disease, which combines a biologic with an enzyme replacement therapy to improve muscle function and quality of life for patients.