If you're looking for rare disease resources and education, you're in the right place.
Due to their, rare diseases often lack the widespread education, awareness, and research attention given to more common conditions. As a result, everyone from patients and their families to policymakers and healthcare professionals may struggle to find reliable information, appropriate diagnostic tools, advocacy resources, and effective treatments.
To bridge this gap, numerous organizations and platforms offer invaluable resources dedicated to rare diseases. Below is a curated list of essential resources that provide comprehensive information, support, and research opportunities for anyone whose life or profession has been touched by rare disease. These resources encompass guides, support groups, databases, clinical research networks, educational materials, patient advocacy groups and information, and more, all aimed at enhancing the understanding, policymaking, and management of rare diseases.
For Nevada-specific resources, please click here.
Cure 4 The Kids Foundation is Southern Nevada’s treatment center for children battling cancer and rare diseases. C4K is dedicated to advancing the treatment and prevention of childhood diseases through clinical excellence and compassionate care.
The Childhood Cancer Hub is a curated, community-driven directory of organizations that support children and teens with cancer, survivors, and their families.
An umbrella organization that brings together smaller foundations focused on orphan diseases. NORD’s site contains excellent information and resources on rare disorders and patient support groups.
A government-run organization dedicated to rare diseases. Their Genetic and Rare Diseases Information Center (GARD) operates under the umbrella of the National Center for Advancing Translational Sciences (NCATS) under the National Institute of Health (NIH). Its aim is to answer questions about rare diseases, share useful information about patient advocacy groups, and to inform about ongoing research trials.
An online network of genetic professionals and patients focused on promoting informational resources and patient advocacy. Genetic Alliance offers many programs, information, and patient-friendly resources.
A European database with high-quality information on rare diseases and orphan drugs, dedicated to gathering and improving knowledge on rare diseases so as to improve the diagnosis, care, and treatment of patients with rare diseases. Orphanet also offers policy reports and recommendations for lawmakers.
A non-profit, nonpartisan organization dedicated to empowering the rare disease patient community to advocate for impactful, science-driven legislation and policy that advances the equitable development of and access to life-saving diagnoses, treatments, and cures.
The Cost of Delayed Diagnosis in Rare Disease: A Health Economic Study
The EveryLife Foundation also offers advocacy tools, legislative scorecards, and policy updates specifically tailored for lawmakers through its RDLA program.
The Undiagnosed Diseases Network (UDN) is a research study funded by the National Institutes of Health (NIH). It seeks to solve challenging medical mysteries and provide answers for patients living with undiagnosed diseases.
Rare Disease Day is February 28th and represents globally-coordinated movement on rare diseases. It raises awareness and supports efforts toward equity in social opportunity, healthcare, access to diagnosis, and therapies for people living with a rare disease.
The Rare Diseases Clinical Research Network (RDCRN) program is designed to advance medical research on rare diseases by providing support for clinical studies, facilitating collaboration, study enrollment, and data sharing.
The Centers for Medicare and Medicaid Services (CMS) is a federal agency that provides information as well as health coverage through Medicare, Medicaid, the Children’s Health Insurance Program, and the Health Insurance Marketplace.
The Global Genes: RARE University offers educational and advocacy resources, toolkits, legislative updates, and webinars for healthcare professionals, advocates, and patients. They also provide policy recommendations to support the creation and implementation of effective rare disease policies.
The Beginner’s Guide to Rare Disease
The European Reference Networks (ERN) offer networks of healthcare providers across Europe aimed at improving the care of patients with rare or low-prevalence complex diseases by pooling expertise and resources.
The National Human Genome Research Institute (NHGRI) is committed to driving the responsible use of genomics in society in order to advance knowledge and ensure that genomics benefits the health of all humans.
And for patients to have full access to the benefits of genetic testing, payers such as insurance companies and Medicare need systematic ways of evaluating genetic tests for reimbursement.
The Center for Drug Evaluation and Research’s (CDER) Accelerating Rare Disease Cures (ARC) program exists to facilitate, support, and accelerate the development of drug and biologic products for the benefit of patients with rare disorders. They also offer guidance on orphan drug development, regulatory information, and resources on rare disease therapies.
A database of privately and publicly funded clinical studies conducted around the world, including those focused on rare diseases.
A detailed guide for diagnosing Guillain-Barre Syndrome (GBS) or Acute Inflammatory Demyelinating Polyradiculoneuropathy (AIDP), Chronic Inflammatory Demyelinating Polyradiculoneuropathy (CIDP), and other variants.
The European Organization for Rare Diseases (EURORDIS) provides policy briefs, position papers, and resources focused on European rare disease legislation, patient rights, and healthcare policies.
The IRDiRC offers resources and strategic plans for rare disease research, fostering international collaboration and informing policy development.
The Global Commission to End the Diagnostic Odyssey for Children with a Rare Disease provides policy recommendations and action plans to improve the diagnosis and care of children with rare diseases.
The Courageous Parents Network provides resources, guidance and community support for parents caring for children with serious illnesses, including rare diseases.
A digital health community where patients and their families share their experiences and find support and information on various health conditions, including rare diseases.
Nevada-specific resources
Based in Las Vegas, Cure 4 The Kids Foundation (C4K) provides specialized medical treatment to children with cancer and rare diseases, offering comprehensive care, support services, and advocacy for patients and their families.
The Nevada Childhood Cancer Foundation (NCCF) offers support services for children diagnosed with life-threatening or rare illnesses, including counseling, financial assistance, educational support, and family activities.
The University of Nevada, Reno School of Medicine provides genomic sequencing and research services, aiding in the diagnosis and understanding of genetic and rare diseases.
The Ackerman Autism Center offers diagnostic, treatment, and support services for children with autism and other developmental disorders, including rare genetic conditions.
The CYSHCN provides resources, support services, and care coordination for children and youth with special health care needs, including those with rare diseases.
The Ronald McDonald House Charities of Greater Las Vegas offers housing and support services to families with children receiving medical treatment for serious or rare conditions in Las Vegas.
Nevada 211 is a comprehensive resource directory that connects Nevada residents to essential health and human services, including those specific to rare diseases.
Nevada PEP provides support, information, and advocacy for parents of children with disabilities, including rare diseases, helping them navigate the state’s education and healthcare systems.
Make-A-Wish Southern Nevada grants wishes to children with critical illnesses, including those with rare diseases, offering emotional and psychological support to families.
Candlelighters provides emotional, educational, and financial support to children diagnosed with cancer and their families, addressing the needs of those affected by rare childhood cancers.
The Little Miss Hannah Foundation supports families caring for children with rare, life-limiting, or complex medical conditions by providing financial assistance, equipment, and family-centered events and support.
The CCSD Homebound Program provides educational services to students who are unable to attend school due to medical conditions, ensuring they continue their education while receiving treatment.
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