#advancing therapies treat

Our Research & Pipeline At Amicus, we don’t just believe that breakthroughs are possible. We believe they are our obligation. There are 10,000 known rare diseases. Together, these diseases affect 300 million people globally, and 25 to 30 million peop

Patient Advocacy Partnering with Patients to Shape Every Step of Care, Discovery, and Progress. At Amicus, the lived experiences of patients guide how we think, act, and innovate — every day. Resources Fabry Disease For information on Fabry disease,

About Us Redefining Expectations in Rare Diseases Amicus is a wholly owned subsidiary of BioMarin Pharmaceutical Inc., a leading, global rare disease biotechnology company focused on delivering medicines for people living with genetically defined con

Hiroki, Age 26 The Wisdom to Focus on What is Good in the Present Moment While Hiroki’s disease may be limiting, his mind is vast and expansive. He is taking nothing for granted. The flowering bushes and cherry blossom trees on the Nagoya University

Kody, Age 43 Wishing for Healing From the Bottom of His Soul “I want this disease to be spoken about in the past tense. That’s what I want for everybody else—for this disease to be a distant memory.” Kody sits up straight against the raised back of h

Lisa, Age 57 Waiting Decades for a Diagnosis “When you go for decades with doctors telling you there is nothing wrong, you think it’s normal—you learn to live with it.” On a misty May morning, Lisa heads out to the backyard to make sure her hostas ha

Our Medicines We’re incredibly proud to have brought forward two treatments for rare diseases for patients in our first two decades, and we’re fully committed to do more.  The information below is intended for use in the United States.   For more inf