Questions about rare diseases?
1-888-205-2311
Questions about rare diseases?
1-888-205-2311
  • Autosomal Dominant Congenital Benign Spinal Muscular Atrophy

You Are Not Alone

Rare diseases are not rare. About 30 million people in the U.S. are affected by a rare disease. As you and your caregivers adjust to a rare disease diagnosis, it is normal to be flooded with a wide range of emotions. Navigating unexpected challenges, coordinating care, and  handling financial concerns may feel overwhelming. GARD recognizes coping with a rare disease diagnosis is a continual process and your needs may change over time. Here, find resources to connect you with practical, financial, and emotional support during this process.
Keep On Fighting

Bringing awareness to rare diseases can lead to funding, research, and treatment. As illustrated in Jacob Thompson's poem "if we work together, there is no impossible task."

Watch the Video

You Are Not Alone

Rare diseases are not rare. About 30 million people in the U.S. are affected by a rare disease. As you and your caregivers adjust to a rare disease diagnosis, it is normal to be flooded with a wide range of emotions. Navigating unexpected challenges, coordinating care, and  handling financial concerns may feel overwhelming. GARD recognizes coping with a rare disease diagnosis is a continual process and your needs may change over time. Here, find resources to connect you with practical, financial, and emotional support during this process.Rare diseases are not rare. About 30 million people in the U.S. are affected by a rare disease. As you and your caregivers adjust to a rare disease diagnosis, it is normal to be flooded with a wide range of emotions. Navigating unexpected challenges, coordinating care, and  handling financial concerns may feel overwhelming. GARD recognizes coping with a rare disease diagnosis is a continual process and your needs may change over time. Here, find resources to connect you with practical, financial, and emotional support during this process.
Keep On Fighting

Bringing awareness to rare diseases can lead to funding, research, and treatment. As illustrated in Jacob Thompson's poem "if we work together, there is no impossible task."

Watch the Video

Patients and Caregivers

Patients and caregivers living with a rare disease may face similar challenges when navigating everyday life. In the next two sections, find tips and resources to become better informed and help you overcome some of these challenges. Learn more about:
  • Financial concerns  
  • Accessing care
  • Navigating health insurance
  • Education and employment
  • Disability services
  • Mental health and self-care
  • Improving care and quality of life

Patients and Caregivers

Patients and caregivers living with a rare disease may face similar challenges when navigating everyday life. In the next two sections, find tips and resources to become better informed and help you overcome some of these challenges. Learn more about:
  • Financial concerns  
  • Accessing care
  • Navigating health insurance
  • Education and employment
  • Disability services
  • Mental health and self-care
  • Improving care and quality of life

Patient Resources

Being diagnosed with a rare disease can place a significant burden on a patient and their caregivers. Impacts can be widespread, affecting finances, access to care, everyday responsibilities, and mental health. The following resources can help address some of these common challenges.

These resources are listed for information purposes only. Inclusion on this list does not reflect an endorsement by GARD or the NIH.

Are There Resources To Help Cover Medical Expenses?

National and regional resources are dedicated to improving access to care and decreasing the financial burdens of a rare disease diagnosis. The Patient Advocate Foundation provides case management assistance for the uninsured or underinsured with life-threatening or debilitating illnesses. Filter by age, location, diagnosis, and/or type of assistance needed to find the right resources.

Explore the National Financial Resource Directory

  • Government programs, low-cost medical and dentals clinics, and prescription assistance are all resources listed by NeedyMeds that may help to cover medical expenses.

  • The National Organization for Rare Disorders (NORD) provides financial assistance for medications, insurance premiums, co-pays, diagnostic testing, and travel for clinical trials or consultations with disease specialists.

  • Community-based fundraising may help offset some of the costs associated with a rare disease diagnosis. Help Hope Live supports community-based fundraising efforts for people with unmet medical expenses and related costs due to illnesses.

Are There Resources To Help With Medical Travel?

  • A number of programs offer medical transportation services, including some that help with travel expenses. NeedyMeds offers a list of these types of resources that can be searched by diagnosis or location.

  • Programs such as Air Care Alliance can help arrange medical transportation, listing available options based on location. Request a flight or information through its website.

  • In some cases, free flights may be available for specific individuals. Patient Airlift Services provides free medical and compassion flights for patients and veterans.

Are There Resources That Assist With Housing During Medical Travel?

  • The Patients Rising concierge maintains a list of discounted lodging for patients and caregivers.

Are There Resources To Help Cover Medical Expenses?

National and regional resources are dedicated to improving access to care and decreasing the financial burdens of a rare disease diagnosis. The Patient Advocate Foundation provides case management assistance for the uninsured or underinsured with life-threatening or debilitating illnesses. Filter by age, location, diagnosis, and/or type of assistance needed to find the right resources.

Explore the National Financial Resource Directory

  • Government programs, low-cost medical and dentals clinics, and prescription assistance are all resources listed by NeedyMeds that may help to cover medical expenses.

  • The National Organization for Rare Disorders (NORD) provides financial assistance for medications, insurance premiums, co-pays, diagnostic testing, and travel for clinical trials or consultations with disease specialists.

  • Community-based fundraising may help offset some of the costs associated with a rare disease diagnosis. Help Hope Live supports community-based fundraising efforts for people with unmet medical expenses and related costs due to illnesses.

Are There Resources To Help With Medical Travel?

  • A number of programs offer medical transportation services, including some that help with travel expenses. NeedyMeds offers a list of these types of resources that can be searched by diagnosis or location.

  • Programs such as Air Care Alliance can help arrange medical transportation, listing available options based on location. Request a flight or information through its website.

  • In some cases, free flights may be available for specific individuals. Patient Airlift Services provides free medical and compassion flights for patients and veterans.

Are There Resources That Assist With Housing During Medical Travel?

  • The Patients Rising concierge maintains a list of discounted lodging for patients and caregivers.

Where Can You Find Help Accessing Care?

  • Health Centers are community-based organizations that deliver comprehensive, culturally aware, high-quality primary health care services to all in need.

  • Access care at a health center funded by the Health Resources and Services Administration (HRSA). You can locate a health center near you by typing your location on this HRSA mapping tool.

  • Find free or reduced-cost health care for lower-income individuals provided by a Hill-Burton facility.

Where Can You Find Guidance On Health Insurance?

  • Find guidance and plan options for health insurance coverage in the United States from HealthCare.gov.

  • The U.S. Government offers a variety of health insurance coverage services. Medicaid is a federal and state health insurance program for people with a low income. The Children’s Health Insurance Program (CHIP) offers health coverage to children. Medicaid and CHIP program names are different in each state.

  • Consumer Assistance Programs (CAP) offer free assistance to help you navigate health insurance options and troubleshoot issues.

Are There Resources To Help Navigate Educational Programs?

  • Parent Centers perform direct services for the families of youth with disabilities. They aim to help parents navigate education and development. Use this tool to learn more and locate your local Parent Center.

  • Understanding how to effectively advocate for the unique needs of your child at their school will help facilitate their educational success. This article guides you through the important steps and considerations when advocating for a child with a rare disease.

  • The Arc advocates for students with intellectual and developmental disabilities to get the school support they need. This nonprofit also provides resources to support students' full inclusion and participation in the community.

  • The CDC offers information on early intervention services that are available to babies and young children with developmental delays and disabilities. Explore who is eligible and where to find programs in each state.

Where Can You Find Guidance For Employment And Disability Benefits?

  • The Job Accommodation Network offers free, one-on-one, and confidential guidance from experts to navigate disability accommodations from employers in the U.S.

  • There are laws in place to protect your right to work for fair pay with the accommodations you need. Learn more about these rights from the National Disability Rights Network.

  • Public assistance programs are available to people who meet certain requirements for disability. The Social Security Administration offers guidance on what to expect during the application process for Social Security Disability Insurance (SSDI) and Supplemental Security Income (SSI).

  • This guide outlines the steps for building and submitting a strong application for public disability benefits.

What If You Are In Crisis?

  • You are not alone. The 988 Lifeline offers free and confidential support for people in distress or in need of emotional support. The service is available 24 hours a day, 7 days a week and is provided by trained Crisis Counselors. Call 9-8-8.

  • The Crisis Text Line is available for any crisis. A live, trained Crisis Counselor receives the text and responds from a secure online platform. Text 741741.

How Can You Support Your Mental Health?

  • An important first step to supporting your mental health is finding a mental health professional to support your needs. Here, find a step-by-step guide to find a professional you feel comfortable talking to. Call 800-950-6264 or text "HelpLine" to 62640.

  • Use this tool to find a psychologist, psychiatrist, therapist, or treatment center near you that accepts your healthcare insurance.

  • Finding a community facing similar challenges can help support your mental health. Learn more about how experiencing chronic illness may impact your mental health. Here, find guidance and resources to locate care in your area.

Caregiver Resources

Caregivers have many responsibilities, often helping with daily life activities, nursing tasks, care coordination, and difficult decision-making. Navigating these responsibilities can cause caregivers to feel overwhelmed or alone. The following organizations, websites, and articles aim to help caregivers organize support and adapt to changing needs.

These resources are listed for information purposes only. Inclusion on this list does not reflect an endorsement by GARD or the NIH.
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Caring for Yourself

Addressing your own needs is not only healthy, it makes you a more effective caregiver. The following websites can help you find ways to create time for yourself, support your emotional health, and connect with others.

How Can You Create Time For Yourself?

  • Programs and local organizations are available to help caregivers take breaks. Find information in this article about the types of programs available to you, and how to overcome the common obstacles in taking time to rest.
  • Find a local respite service that provides temporary care while you take breaks. This page provides a directory of respite care and lists tips for finding adult day care centers, summer camps, crisis nurseries, and other services.

How Can You Support Your Emotional Health?

  • It is important to continue to tend to your own needs while caring for a loved one. Here, find advice for balancing your own well-being as a caregiver.

How Can You Connect With Other Caregivers?    

  • Find peer support by connecting with other caregivers. Give an Hour facilitates expert-led training programs and offers specialized caregiver resources.
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Caring for Others

As a caregiver, it may be challenging to find the support and resources you need to provide proper care to others. The following resources provide information and tools on home health care support, optimizing your home for caregiving, and building a community of support.

Where Can You Find Home Health Care Support?

  • There are services available to assist you in caring for your loved one, whether they live at home or in a residential facility. Here, find resources like health and disability programs, disease-specific organizations, and legal resources local to you.

How Can You Optimize Your Home for Caregiving?

  • Modifying your home environment to suit your caregiving needs can be expensive. Disability grants and programs can assist with implementing home improvements and repairs. This list details a few options that may help reduce your out-of-pocket costs.

How Can You Build a Community of Support?

  • There is a lot to balance when organizing care, meals, and other support for a loved one. This free app helps you coordinate support and provides a platform to keep others updated on needs and health changes.
  • This free app was designed for caregivers to organize documents, schedules, and prescription information, communicate needs, and coordinate help.

Making Informed Health Care Decisions

As you navigate supplemental care options, it is important you make informed health care decisions to ensure you receive the care you want and need. For example, you may consider exploring alternative types of care alongside traditional medicine, or you may be interested in connecting with patient-centered organizations that focus on enhancing health care quality. To make informed health care decisions at every stage of your journey, it is important to know what care options are available, and where to find reliable resources.
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Exploring Complementary Care

Complementary care refers to medical products and practices that may be used alongside traditional medicine to treat your disease or related symptoms. For example, complementary care treatments may include nutritional supplements, physical massage, or meditation. It is important to consider multiple factors when examining the use of complementary care, such as access to a provider, the available resources, and potential out-of-pocket costs.

How do you find accurate and reliable information?

  • Understanding how to find and evaluate information about your health online can help guide your search for complementary care resources. Knowing the science behind your health can help you better evaluate available resources. 
  • Online tools can help you determine which resources provide accurate information on complementary and integrative health approaches. Recognizing which resources you can trust can help you find the correct combination of care for your disease.

What should you consider when evaluating complementary care?

  • Finding the right practitioner is critical to creating a complementary care plan that works for you. Understanding how to evaluate a practitioner's experience treating your disease can help you find the most effective course of care.
  • The use of complementary care practices may come with unexpected expenses. Being aware of what your insurance will or will not cover may help you to determine which complementary care practices to integrate into your treatment plans.

How can you learn more about alternative treatments used in complementary care?

  • Resources on non-traditional health care options highlight a variety of topics. To learn more about alternative treatments, you may want to explore topics ranging from restorative massage to functional food. 
  • Alternative health care treatments can often include the use of herbs and botanicals, also known as plant-based additives. These fact sheets include common names, background information, scientific information, and additional resources.

Exploring Complementary Care

Complementary care refers to medical products and practices that may be used alongside traditional medicine to treat your disease or related symptoms. For example, complementary care treatments may include nutritional supplements, physical massage, or meditation. It is important to consider multiple factors when examining the use of complementary care, such as access to a provider, the available resources, and potential out-of-pocket costs.

How do you find accurate and reliable information?

  • Understanding how to find and evaluate information about your health online can help guide your search for complementary care resources. Knowing the science behind your health can help you better evaluate available resources. 
  • Online tools can help you determine which resources provide accurate information on complementary and integrative health approaches. Recognizing which resources you can trust can help you find the correct combination of care for your disease.

What should you consider when evaluating complementary care?

  • Finding the right practitioner is critical to creating a complementary care plan that works for you. Understanding how to evaluate a practitioner's experience treating your disease can help you find the most effective course of care.
  • The use of complementary care practices may come with unexpected expenses. Being aware of what your insurance will or will not cover may help you to determine which complementary care practices to integrate into your treatment plans.

How can you learn more about alternative treatments used in complementary care?

  • Resources on non-traditional health care options highlight a variety of topics. To learn more about alternative treatments, you may want to explore topics ranging from restorative massage to functional food. 
  • Alternative health care treatments can often include the use of herbs and botanicals, also known as plant-based additives. These fact sheets include common names, background information, scientific information, and additional resources.

Transitioning Care

Health care transitions are common between childhood and adulthood but can occur at any stage in life. These transitions can be complicated, especially for those with a rare disease, because gaps in care can sometimes lead to major health issues.

Is Your Child Transitioning From Pediatric to Adult Care?

  • For young adults with rare diseases, guidance and financial advocacy resources can simplify the transition from pediatric to adult care. This article includes information on making a transition plan, finding a primary care provider, patient advocacy, navigating insurance plans, managing care, and living alone.
  • Understanding the timeline when moving from pediatric to adult care can be challenging. Having resources and clearly outlined steps to asses your readiness for this process make the transition more manageable. Here, find a guide for this care transition and resource pages from health care and advocacy organizations. Resources are organized by intended audience, including youth and young adults, parents and caregivers, and health care providers.

Does Your Child Have Special Health Care Needs?

  • Some individuals may benefit from specialized support and resources for school-to-adult life transitions. On this page, find resources for families of children aged 12 to 26 that offer guidance for using tools like telehealth during care transitions.
  • For individuals ages 10-26 with special health care needs, these guides to health care transitions, life skills, and career planning may be useful. Guides are arranged by age range and life stage to assist in a successful transition from pediatric to adult care.

Palliative and Hospice Care

Patients living with a serious illness or entering the end stages of life may need specific additional support, known as palliative care or hospice care. Palliative care focuses on addressing symptoms and reducing stress for those with serious illnesses. Hospice care provides care and comfort for those approaching the end of life.

How Can You Learn More About Palliative and Hospice Care?

  • When considering palliative care you may have concerns related to logistics, effectiveness, and what care is included. To learn more about palliative care, review the evidence-based answers linked below to common questions about how palliative care can support patients, families, health care providers, and communities.
  • While the goal of both palliative and hospice care is to improve patients' quality of life, they address different health care needs. Compare the similarities and differences between palliative care and hospice care to learn which is best suited to your situation.
  • Explore resources for patients and caregivers curated to help make informed decisions about serious illness and end-of-life care. This web page provides information on how to prepare for care, descriptions of the different types of care, and resources for patients and caregivers.
  • Understanding the process of connecting with a provider and what to expect from palliative care will help you decide if it is right for your situation. Here, find resources to determine if palliative care is needed, and a Provider Directory to help find care where you live. Information is available in English and Spanish.

Are You Preparing for Hospice Care?

  • For individuals who have decided hospice care is appropriate for their health needs, there are many logistical questions to answer. Learning more about how to get started accessing care, how care can be paid for, where to find providers, and what care is included in hospice can help you prepare.

Accessing Investigational Treatments

Many rare diseases are limited in their treatments or have no treatments at all. In some cases, treatments are still under investigation or haven't been researched in relation to your specific rare disease. Expanded access and off-label use are two possible methods of gaining access to these investigational treatments when other treatments are not available. Consult your medical team before participating in these treatment options as they may come with elevated risk.

What is expanded access?

  • Expanded access may be an option to consider if all treatment options have been explored or if there are no other available treatment options. Ask yourself: Who can request expanded access? How do you request it? When is expanded access appropriate? These questions and more may help guide your exploration of expanded access.
  • Expanded access is also referred to as compassionate use. Here, explore answers to frequently asked questions on expanded access along with information on clinical studies, guidance documents, program data, and learning material.
  • Expanded access may involve navigating through different regulations, increased risks/costs, and potential emotional impacts. It is important to review resources covering a wide range of topics related to expanded access before using this treatment option.

What is off-label use?

  • Off-label use involves using U.S. FDA-approved drugs to treat conditions that the drug is not yet approved for. Here, learn why off-label use occurs, examples of off-label use, and questions you should ask yourself before choosing this treatment option.
GARD Information Specialists can help you find resources focused on living with this disease. 

Please contact GARD if you need additional assistance.
1-888-205-2311
Available toll-free Monday through Friday from 12 pm to 6 pm Eastern Time
(Except: Federal Holidays)
Use the contact form to send your questions to a GARD Information Specialist.

Please allow 2 to 10 business days for us to respond.
GARD Information Specialists can help you find resources focused on living with this disease. 

Please contact GARD if you need additional assistance.
1-888-205-2311
Available toll-free Monday through Friday from 12 pm to 6 pm Eastern Time
(Except: Federal Holidays)
Use the contact form to send your questions to a GARD Information Specialist.

Please allow 2 to 10 business days for us to respond.

Learn about symptoms, cause, support, and research for a rare disease. 

Take steps toward getting a diagnosis by working with your doctor, finding the right specialists, and coordinating medical care.

Find resources for patients and caregivers that address the challenges of living with a rare disease.

Last Updated: February 2023