Advocacy Groups

As a key influencer and mobilizer of the patient community, advocacy groups play a critical role in supporting and promoting the needs of people affected by rare diseases. While some groups are highly organized associations representing thousands of patients, others may be small, one-person volunteer operations that focus primarily on patient care and support. Regardless of size, each organization plays a critical role in supporting the patient community and has the ability to influence the wider rare disease community and represent the needs of their members to industry, the media, and government and policy makers.

Patient Organizations

Following are lists of the many organizations with which we are most familiar, or with whom we most frequently collaborate, especially within the communities of lysosomal storage disorders.

Disease-Specific Groups

Disease-specific groups aligned with the rare disorders on which Genzyme focuses: Gaucher disease, Fabry disease, Familial Hypercholesterolemia, Pompe disease, MPS I, Thyroid Cancer.

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Fabry Disease

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Familial Hypercholesterolemia (FH)

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Gaucher Disease

  • ARGENTINA: Asociación Gaucher Argentina / Gaucher Association Argentina
    www.gaucher.com.ar
  • BRAZIL: Associação Brasileira dos Portadores da Doença de Gaucher / Brazilian Association of Gaucher Disease)
    www.appdgaucher.org.br
  • CANADA: The National Gaucher Foundation of Canada (NGF Canada)
    www.gauchercanada.ca/

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Mucopolysaccharidosis I (MPS I)

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Niemann-Pick Disease

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Pompe Disease

  • AUSTRALIA: Australian Pompe Association
    www.australianpompe.com
  • GLOBAL: International Pompe Association (IPA)
    www.worldpompe.org
  • U.K.: Muscular Dystrophy Campaign
    www.muscular-dystrophy.org
    Although not a Pompe-specific organization, the Muscular Dystrophy Campaign advocates for a range of neuromuscular disorders, including Pompe disease.
  • U.S.: Acid Maltase Deficiency Association (AMDA)
    www.amda-pompe.org
  • U.S.: Association for Glycogen Storage Disease
    www.agsdus.org
  • U.S.: United Pompe Foundation
    www.unitedpompe.com
  • U.S.: Muscular Dystrophy Association (MDA)
    www.mdausa.org/
    Although not a Pompe-specific organization, the MDA advocates for a range of neuromuscular disorders, including Pompe disease.
  • U.S.: The Children's Cardiomyopathy Foundation (CCF)
    www.childrenscardiomyopathy.org
    Although not a Pompe-specific organization, the CCF advocates for other disease like Pompe that may involve cardiac complications.

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Thyroid Cancer

Allied Health Groups

Allied Health Groups cover a variety of diseases, although most focus on a specific category such as rare, genetic, or metabolic disorders.

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