Living with Pompe Disease
Welcome to Amicus Patient & Professional Advocacy
Amicus Patient & Professional Advocacy collaborates with patient organizations, individual patients, their caregivers, and healthcare practitioners to ensure we are doing all that we can to help support the rare disease community.
We will continue to provide initiatives that bring purpose and empowerment to help ease the difficulty that members of the rare disease community experience. We are dedicated to being the bridge between patients, their families, and resources, offering access to support and services across their disease experience. Our dedication to patients and their families is what drives us to truly provide healing beyond disease. Our passion drives our commitment to members of the rare metabolic disorder community by:
- Listening and learning from individuals and their families affected by these diseases;
- Searching for new and better therapies;
- Incorporating patient input into the design of clinical trials for those therapies as our research advances through the drug development process; and
- Providing useful information and resources to help manage the daily challenges of living with a rare, genetic disease.
Jayne C. Gershkowitz
Chief Patient Advocate
We established the Patient Advisory Board (PAB) Program in 2008 to enhance our partnership with and understanding of the patient community
Amicus is committed to carefully designed and considered paths to Expanded Access to our investigational drugs.
Access resources and helpful links for patients and families living with Fabry disease, Pompe disease, Batten disease, CDKL5 deficiency and other conditions.