These resources offer helpful information to support a wide scope of audiences, including patients, caregivers, advocates, researchers, and health care providers:

• Global Genes is a non-profit organization dedicated to eliminating the burdens and challenges of rare diseases for patients and families globally.

• National Organization for Rare Diseases a nonprofit organization that promotes research, advocacy, awareness, and public policies that benefit rare disease communities nationwide.

• NORD Center of Excellence-Georgia
  https://rarediseases.org/center-of-excellence/emory-division-of-medical-genetics-childrens-healthcare-of-atlanta/

• Newborn Screening: The Newborn Screening Program, managed by the Georgia Department of Public Health, allows for the testing of every newborn in GA to check for harmful disorders that aren’t otherwise apparent at birth. This GA DPH website offers manuals for and information about the disorders for which the testing screens, as well as information sheets on the various disorders for both medical providers and parents/caregivers.

• Rare Advocacy Movement represents a rare disease patient and carer ecosystem that relies upon specialized global collaborations to address the needs of the patient and carer community from the community perspective.

• Rare Diseases Clinical Research Network is designed to advance medical research on rare disease by providing support for clinical studies and facilitating collaboration, study enrollment, and data sharing.

• Rare Disease Day is held annually on the last day of February to raise awareness and promote research and advocacy for the rare disease community.

• Rare Disease Diversity Coalition unifies rare disease experts, health and diversity advocates, and industry leaders to identify and advocate for evidence-based solutions to alleviate the disproportionate burden of rare diseases on historically marginalized populations.

• Rare Wish -mission is to improve the quality of life for rare families by granting wishes, funding clinical innovation, and offering free healthcare tools.

• Rare STRIDES -mission is to empower patients and medical teams with innovative tools to successfully fight rare diseases, and we are committed to the delivery of exceptional clinical care for rare patients in every medical setting. We are passionate about giving back to fund patient programs and continued innovation for children and adults with rare diseases who deserve excellence in healthcare.

In addition to these organizations, the Georgia Rare Disease Advisory Council has identified additional online resources that they have organized for patients, advocates, providers, and researchers, and categorized according to topic areas relevant to those specific groups.

We also encourage you to visit our Research page if you are seeking clinical research studies to learn about and participate in as a volunteer.

OMIM-online mendelian inheritance of man. An online catalogue of human genes and genetic disorders.

Clinical Trials-an online database of clinical research studies and information about their results posted by the NIH National Library of Medicine

Medscape-the latest news and journal articles on rare diseases.

PubMed-PubMed® comprises more than 37 million citations for biomedical literature from MEDLINE, life science journals, and online books. Citations may include links to full text content from PubMed Central and publisher web sites.

Genetic and Rare Disease Information-National Institute of Health center for information and resources on rare diseases.

These resources are intended to benefit patients, advocates, and caregivers at any given stage of their rare disease journey: from seeking an official diagnosis and learning about rare disease symptoms and treatments, to managing the costs of healthcare, to promoting advocacy and awareness.

• Undiagnosed Diseases Network is an NIH supported research study that seeks to provide answers for patients and families affected by these mysterious conditions.

• Our Odyssey-A community by and for young adults impacted by a rare or chronic condition.

• #Rareis-Elevating the voices, faces, and experiences of people living with rare disease.

• Rare Wish -mission is to improve the quality of life for rare families by granting wishes, funding clinical innovation, and offering free healthcare tools.

• EveryLife Foundation-a nonprofit, 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 lifesaving diagnoses, treatments and cures.

• National Organization for Rare Diseases a nonprofit organization that promotes research, advocacy, awareness, and public policies that benefit rare disease communities nationwide.

• Canine Companions for Independence-a non-profit organization that enhances the lives of people with disabilities by providing expertly trained service dogs and ongoing support to ensure quality partnerships.

• Good Days-a national non-profit charitable organization that lifts the burdens of chronic illness through assistance, advocacy, and awareness.

• ARCH-national respite network and resource center that helps caregivers rest and recharge.

• Caregiver Action Network-toolbox full of caregiver resources.

• Courageous Parents Network- a nonprofit organization that has oriented, equipped and empowered those caring for children with a serious medical condition.

• NCATS Genetic and Rare Diseases Information Center -GARD is a program of the National Institutes of Health that provides free access to information about genetic and rare diseases.

• NORD Rare Disease Database-The NORD Rare Disease Database is an inclusive and comprehensive list of rare diseases based on information from established rare disease ontologies and it is an ongoing project made possible by a close collaboration with MONDO, Orphanet, and Online Mendelian Inheritance in Man (OMIM).

• American Academy of Pediatrics-disaster preparedness resources for families.

• Disability Resources-preparing for emergencies and disasters.

• Disaster Safety for People with Disabilities-planning ahead for emergencies.

• How to Prepare for Emergencies-resources prepared by the American Red Cross

• NephCure-helps provide access to new treatments, including more than 60 interventional drug trials for rare kidney disease.

Rare Disease Advisor-rare disease news, perspectives, and resources.

Medscape-the latest news and journal articles on rare diseases.

PubMed-PubMed® comprises more than 37 million citations for biomedical literature from MEDLINE, life science journals, and online books. Citations may include links to full text content from PubMed Central and publisher web sites.

EveryLife-CostofDelayedDiagnosis-the EveryLife Foundation for Rare Diseases is a nonprofit organization that empowers the rare disease patient community to advocate for impactful, science-driven legislation and policy. Their goal is to advance the equitable development of and access to lifesaving diagnoses.

Rare Disease Diversity Survey-report on the survey results regarding inequities in the Rare Disease Community published in 2024.

The Assistance Fund

Patient Advocate Foundation Copay Relief

Patient Assistance Program NORD

Patient Assistance Rare Wish

• Auto Immune is a global nonprofit focused on promoting autoimmune disease awareness, advocacy, education and research.

• NeedyMeds is a non-profit dedicated to improving access to affordable healthcare for individuals in need.

• Patient Advocate Foundation provides patient services and promotes health care equity by removing obstacles to quality health care access.

• RARECARE Patient Assistance Programs provides information on caregiver aid, financial assistance, and educational support for patients and caregivers of patients with rare diseases.

• Cure VCP Disease-nonprofit uncompromising in their dedication to finding a cure for VCP disease and giving hope to the many families worldwide impacted by this devastating disease.

• Rare Wish -nonprofit mission is to improve the quality of life for rare families by granting wishes, funding clinical innovation, and offering free healthcare tools.

• National Organization for Rare Diseases a nonprofit organization that promotes research, advocacy, awareness, and public policies that benefit rare disease communities nationwide.

• Global Genes is a non-profit organization dedicated to eliminating the burdens and challenges of rare diseases for patients and families globally.

• EveryLife Foundation-a nonprofit, 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 lifesaving diagnoses, treatments and cures.

• Canine Companions for Independence-a non-profit organization that enhances the lives of people with disabilities by providing expertly trained service dogs and ongoing support to ensure quality partnerships.

• Good Days-a national non-profit charitable organization that lifts the burdens of chronic illness through assistance, advocacy, and awareness.

• ARCH-national respite network and resource center that helps caregivers rest and recharge.

• Caregiver Action Network-toolbox full of caregiver resources.

Courageous Parents Network- a nonprofit organization that has oriented, equipped and empowered those caring for children with a serious medical condition.

Rare Disease Research-an independent clinical research site, 100% dedicated to conducting clinical research, accelerating the development of safe and effective treatments for rare diseases, and providing access to innovative investigational therapies to patients with rare diseases.

Clinical Trials-an online database of clinical research studies and information about their results posted by the NIH National Library of Medicine.

Research Match-is made up of volunteers, researchers, and community collaborators working together to make it easy for you to find the right clinical trial or research study for you or your family.

• IAMRARE Registry is an online registry on NORD’s platform that helps patients share data and researchers begin studies that will inform medical research and translational science for rare diseases.

• Genome Connect is an online registry that helps patients share their genetic and health information.

• Miracle Flights-provides free commercial flights to children in need of life changing medical care not found in their local communities.

• Angel Flight-Since 1983, volunteer pilots have been flying patients to their life-changing medical treatments, free of charge, giving hope to families in crisis.

• Mercy Medical Angels-removes the barrier to medical care with transportation on the ground and in the air.

• PALS-Patient AirLift Services is a nonprofit that arranges free flights for medical patients requiring medical diagnosis, treatment or follow-up who cannot afford or are unable to fly commercially.

Managing Financial Costs
Managing the cost of rare disease diagnosis and treatment can be expensive and challenging for patients and their families. The following resources are available for those who qualify for financial assistance:

Federal Resources

• COBRA is government legislation that gives workers and families who lose their benefits through job loss the opportunity to elect continuing group health benefits provided by their employer for a limited time period.

• Medicare is a government insurance program designated for people 65 years of age or older, young people with disabilities, and people with end-stage renal disease.

• Medicaid is a government insurance program for families and individuals with low income.

• Affordable Care Act offers access to the government insurance marketplace for health insurance policies which are provided by private insurance companies for individuals, families, and small businesses.

• USA.Gov provides links to federal resources that offer financial assistance for medical costs and prescription drugs.

State Resources

• Georgia’s Medicaid program that provides health coverage for eligible SC residents.

• GA Department of Social Services offers assistance programs to families who are eligible to receive financial assistance.

• GA Health Insurance Assistance Program provides help and resources for aging GA residents with disabilities.

These resources are intended to support clinicians and researchers who are seeking support at various stages of their clinical practices and research studies: from identifying current diagnosis and treatment methodologies for rare disease patients, to collaborating on research and registry programs, to learning how to improve patient treatment and care.

• OMIM-online mendelian inheritance of man. An online catalogue of human genes and genetic disorders.

• Human Phenotype Ontology-The Human Phenotype Ontology (HPO) provides a standardized vocabulary of phenotypic abnormalities encountered in human disease.

• Clinical Trials-an online database of clinical research studies and information about their results posted by the NIH National Library of Medicine.

• Medscape-the latest news and journal articles on rare diseases.

• PubMed-PubMed® comprises more than 37 million citations for biomedical literature from MEDLINE, life science journals, and online books. Citations may include links to full text content from PubMed Central and publisher web sites.

• Genetic and Rare Disease Information-National Institute of Health center for information and resources on rare diseases.

• NCATS Genetic and Rare Diseases Information Center is an NIH supported institute that offers resources and toolkits for research, funding, and training for researchers.

• NORD Clinicians and Researchers is a network powered by NORD to provide support in research and health care for clinicians and researchers of rare disease.

Novel diagnostics and therapeutics for rare diseases can only be developed through the conduction of clinical research and participation of research volunteers. These clinical trials are monitored by the NIH and U.S. FDA. Whether you are seeking to stay current in medical conditions and research studies, collaborate on registry programs, or learn how to improve patient treatment and care, then there are several resources available.

• C-Path is a nonprofit that works with the FDA under the 2005 Critical Path Initiative Program to foster collaboration between industry leaders, regulators, researchers, scientists, and patients to help improve and streamline the drug development process for people with critical medical needs.

• The Georgia Clinical & Translational Science Alliance leverages its institutions complementary strengths to accelerate clinical & translational education, research, & community engagement to impact health.

• Rare Diseases Registry Program helps researchers learn how to build and manage their registries in order to connect with patients of the rare disease community.

• RDCRN Research Group is an NIH-funded research network that encourages collaborative research to better understand and improve diagnosis and treatment of rare diseases.

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Georgia RDAC Publishes Its 2024-2025 Annual Report: Building Momentum for Georgia’s Rare Disease Community
Georgia’s Rare Disease Advisory Council (RDAC) has released its inaugural Annual Report, covering activities from April 2024 through June 2025. Established by law on May 6, 2022, and fully seated in early 2024, the Council’s mission is to ensure every Georgian living with a rare disease has timely diagnosis, coordinated care, evidence-based resources, and access to effective treatments.Key Highlights

• Operational Launch & Governance

First official meeting in April 2024; Council now meets monthly and is guided by adopted bylaws, a mission statement, and three strategic goals.

Diverse 2025 membership of clinicians, patient advocates, researchers, industry, and state-agency representatives brings cross-sector expertise to the table.

• Strategic Goals & Early Wins

1. Policy Resource:

• Engaged state legislators and multiple agencies, delivered testimony, and supplied technical feedback on rare-disease legislation during the 2025 session.

2. Empower the Rare Community:

• Drafted Georgia’s first Quality of Life Needs Assessment Survey (to launch in 2026) and attended community events such as Rare Wish and NORD’s conferences to elevate patient voices.

3. Support the Medical Community:

• Provided guest lectures at Emory Medical School, the GA Bio Patient Engagement Summit, and other venues to raise clinical awareness of rare diseases.

• Outreach & Education

• Hosted nationally recognized speakers from NORD, PhRMA, and academic researchers to share best practices and policy insights with Council members.

• Launched a new RDAC website, improving public access to resources and Council updates.

• Metrics at a Glance10 direct support requests from rare-disease patients or groups answered.
  3 patient testimonials shared with policymakers.
  2 community events attended and 3 listening-style presentations delivered.
  2 bills supported or shaped through Council engagement.

Looking Ahead to 2026
The Council will broaden its impact by hosting statewide listening sessions, finalizing and deploying the Quality of Life Survey, expanding collaborations with healthcare providers, and solidifying its role as a trusted policy resource for Georgia’s General Assembly.

“United, we will help our rare community thrive for years to come.” — Beth Nguyen, RN, RDAC ChairpersonRead the full 2024-2025 Annual Report to see detailed accomplishments, financials, and the complete roadmap for advancing rare-disease care and research in Georgia.