Resources

Looking for resources and additional information on the diseases within our areas of focus? Look no further.

Rare Diseases

  • Global Genes – Allies in Rare Disease
    Global Genes is a leading rare disease patient advocacy organization whose mission is simple: to eliminate the challenges of rare disease. They do this through developing educational resources, providing critical connections, and equipping advocates to become successful activists.

    National Organization for Rare Disorders (NORD)
    The National Organization for Rare Disorders (NORD), a 501(c)(3) organization, is a unique federation of voluntary health organizations dedicated to helping people with rare orphan diseases and assisting the organizations that serve them. NORD is committed to the identification, treatment, and cure of rare disorders through programs of education, advocacy, research, and service.

  • Immune Deficiency Foundation (IDF)
    The Immune Deficiency Foundation (IDF) is the national patient organization dedicated to improving the diagnosis, treatment, and quality of life of persons with primary immunodeficiency diseases, such as CGD, through advocacy, education, and research. IDF provides accurate and timely information and resources to patients and healthcare professionals.

    Jeffrey Modell Foundation
    JMF is a global nonprofit organization dedicated to early diagnosis, meaningful treatments and, ultimately, cures for primary immunodeficiency diseases through research, physician education, public awareness, advocacy, patient support, and newborn screening. The Jeffrey Modell Centers Network (JMCN) includes over 600 expert physicians at 250 academic institutions, in 206 cities, 84 countries, spanning 6 continents and growing.

    HorizonCares™ Program
    The HorizonCares program emphasizes Horizon Pharma’s commitment and willingness to go the extra mile for all the patients we serve, with services and support that assist patients living with rare diseases and their caregivers. An important part of this commitment is ensuring that eligible patients have access to medicines prescribed by their healthcare providers. We understand that some people have difficulty paying for their prescriptions and our goal is to help limit their financial burden. We offer comprehensive assistance programs that provide eligible patients with a range of support services, co-pay assistance and access to medicines for free or at a saving.

  • Ryan Wersten MIOP Foundation
    The Ryan Wersten MIOP Foundation was created in memory of Ryan Wersten who lived for only six months after being diagnosed with Malignant Infantile Osteopetrosis (MIOP), also known as severe, malignant osteopetrosis. The Ryan Wersten MIOP Foundation is dedicated to supporting current research and to supporting families who have a child with MIOP.

    Mason Shaffer Foundation
    The Mason Shaffer Foundation is a nonprofit organization that has been dedicated to creating a support network for families of children with osteopetrosis.

    The OsteoPETrosis Society (OPETS)
    The OPETS is an organization that provides education and support to people diagnosed with osteopetrosis and medical professionals dealing with the disease. The Society also conducts informative sessions for patients through a Medical Advisory Council that provides expertise in the field of osteopetrosis.

  • National Urea Cycle Disorders Foundations (NUCDF)
    The National Urea Cycle Disorders Foundation is a non-profit organization dedicated to the identification, treatment and cure of urea cycle disorders. NUCDF is a nationally-recognized resource of information and education for families and healthcare professionals. It was formed in 1988 by a handful of parents whose children were affected. The Foundation is operated and supported by the volunteer efforts of families with children suffering from UCDs and is a supportive network of families, friends, and medical professionals.

    Connecting Families UCD Foundation (CFUCDF)
    Connecting Families UCD Foundation is dedicated to bringing together those affected with Urea Cycle Disorders by empowering and engaging those affected all over the world. We strive to educate, bring awareness, and provide critical tools needed to improve the lives of all those with this rare disease.

    It is our hope to one day see that a cure is found, but until then we will do our best to improve the lives of our UCD friends and families.

    Urea Cycle Disorders Consortium (UCDC)
    The Urea Cycle Disorders Consortium is a team of doctors, nurses, research coordinators, and research labs throughout the US, working together to improve the lives of people with Urea Cycle Disorders. Since Urea Cycle Disorders are rare, there are low numbers of research participants and this adds a greater level of difficulty for researchers struggling to learn more about these disorders. The purpose of this consortium is to provide a way for patients to join with doctors and researchers by participating in research studies. The greater the collaboration between doctors and patients, the more we can learn about Urea Cycle Disorders. This important first step is necessary if we are ever to find newer treatments.

  • Cystinosis Foundation
    The Cystinosis Foundation is a non-profit organization with more than 30 years of International experience in supporting and educating families and the medical community through the dissemination of educational literature, funding research, and annual conferences.

    Cystinosis Research Foundation
    The Cystinosis Research Foundation is dedicated to finding better treatments to improve the quality of life for those with cystinosis and to ultimately find a cure for this devastating disease. Since 2003, it has raised and committed more than $35 million to support bench, clinical and translational research to find better treatments and a cure for cystinosis. The foundation is dedicated to educating the public and medical community about cystinosis to ensure early diagnosis and proper treatment.

    Cystinosis Research Network
    The Cystinosis Research Network is an all-volunteer, non-profit organization with the mission of providing family assistance and educating the public about cystinosis. The organization also supports and advocates for research with the hope of accelerating the discovery of a cure and development of improved treatments to enhance the quality of patients’ lives.

Rheumatic Disease Resources

  • The Arthritis Foundation
    The Arthritis Foundation is the largest national nonprofit organization that supports the more than 100 types of arthritis and related conditions. The Arthritis Foundation offers information and tools to help people live a better life with arthritis.

    CDC’s Arthritis Website
    Information and resources within the arthritis section of the U.S. government’s Centers for Disease Control and Prevention (CDC) website.

    ACR Simple Tasks
    Simple Tasks is a public awareness campaign by the American College of Rheumatology that strives to increase awareness of rheumatic diseases and the work of rheumatologists, as well as implement favorable public policy in the United States. Simple Tasks aims to answer common questions about arthritis and other rheumatic diseases. By increasing the visibility of rheumatic diseases, how they affect millions of patients and the role of the specialists who treat them, the ACR hopes influencers will recognize the value, understand the issues, and make decisions that support rheumatology and the patients served by the rheumatology health care team.

Gout Resources

  • Gout and Uric Acid Education Society (GUAES)
    The Gout & Uric Acid Education Society is a nonprofit organization of health care professionals dedicated to educating the public and health care community about gout – an extremely painful form of inflammatory arthritis – and the related health care consequences of hyperuricemia. With the aim of improving the quality of care and minimizing the burden of gout, the Gout & Uric Acid Education Society offers complimentary resources for both the public and medical professionals.

    Arthritis Foundation: Wipe Out Gout
    Wipe Out Gout is a toolkit created by the Arthritis Foundation to help raise awareness of gout, and the fact that zero flares of the sudden, excruciating pain known as “gout flare” may be possible. The toolkit also includes tips and tools on how to talk to your doctor about gout.

    CreakyJoints
    CreakyJoints is the world’s leading arthritis support, education, advocacy and research community for people with arthritis (rheumatoid arthritis, psoriatic arthritis, osteoarthritis, and other rheumatic diseases) as well as their caregivers. It is a part of the non-profit Global Healthy Living Foundation (GHLF). GHLF and CreakyJoints work to create an environment where strength, experience, and information can be exchanged to improve quality-of-life through educational programs, supportive social media, innovative research initiatives and nurtured collaborative advocacy among other organizations. Membership within CreakyJoints is free and encouraged for anyone who is impacted by arthritis.