At Applied Therapeutics, the patient advocacy team is passionate about educating and supporting patients, families and caregivers. Our organization applauds the patient advocacy organizations dedicated to supporting people living with serious illnesses. We focus on listening, understanding and representing the views of patients so they are heard and valued.

As representatives of the Applied Therapeutics Patient Advocacy team, our company follows the PhRMA Principles on Interactions with Patient Organizations and the BIO Guiding Principles for Interaction with Patient Advocacy Organizations.

If you have any questions, please email Dottie Caplan at

Rare Disease Organizations

  • National Organization for Rare Disorders (NORD)
    NORD is a patient advocacy organization dedicated to individuals with rare diseases and the organizations that serve them. NORD is committed to the identification, treatment, and cure of rare disorders through programs of education, advocacy, research, and patient services.
  • Global Genes
    Global Genes is a global nonprofit advocacy organization for individuals and families fighting rare and genetic diseases.
  • EveryLife Foundation
    EveryLife Foundation is dedicated to advancing the development of treatment and diagnostic opportunities for rare disease patients through science-driven public policy.

Galactosemia Organizations

  • Galactosemia Foundation (GF)
    GF (formerly, Parents of Galactosemic Children) is a nonprofit charitable organization that advocates for people with Galactosemia and their families.


  • Charcot-Marie-Tooth Association (CMTA)
    CMTA is devoted to supporting the development of new drugs to treat CMT, improving the quality of life for people with CMT, and, ultimately, finding a cure.
  • Hereditary Neuropathy Foundation (HNF)
    HNF is a nonprofit organization whose mission is to increase awareness and accurate diagnosis of Charcot-Marie-Tooth (CMT) and related inherited neuropathies, support patients and families with critical information to improve quality of life, and fund research that will lead to treatments and cures.

PMM2-CDG Organizations

Heart Health Organizations

  • American Heart Association (AHA)
    The AHA is a nonprofit organization that funds cardiovascular medical research, educates consumers on healthy living and fosters appropriate cardiac care to reduce disability and deaths caused by cardiovascular disease and stroke.
  • Mended Hearts
    Mended Hearts is a national and community-based nonprofit organization that has been offering the gift of hope to heart disease patients, their families and caregivers.
  • WomenHeart
    WomenHeart is the leading voice for the 48 million American women living with or at risk of heart disease.

Diabetes and Neuropathy Organizations

  • American Diabetes Association (ADA)
    The ADA is a network of more than 565,000 volunteers, a membership of more than 540,000 people with diabetes and their families/caregivers, a professional society of nearly 20,000 health care professionals, and has more than 400 staff members.
  • The diaTribe Foundation
    The diaTribe foundation wants to make life happier and healthier for people living with diabetes, prediabetes, and obesity. They advocate for recognizing the emotional impact that diabetes has, as well as collaboration across government, nonprofits, and the healthcare industry.
  • DiabetesSisters
    DiabetesSisters was founded in response to a need for more education and advocacy around the health of women with diabetes.

General Information on Clinical Trials