A disease may be rare, but hope should not be.

You are not alone.

This page provides resources and information to help navigate your GM1 journey. Whether you’re a parent of a newly diagnosed child or you have been a caregiver for a while, we hope this information can be of assistance.

letter to New Families

Dear parent and caregiver, if you found us, you must be in search of help and information. You might be feeling overwhelmed and a bit lost. Take a breath, you found your community, and we’re here to help you.

We are working each and every day to advance research for GM1, and promising treatments are being developed for GM1, such as small molecules, to gene therapies, and enzyme replacement therapies. There are many projects which lack funding and a dedicated team. Possible treatments already exist, but they have been shelved or are out of reach.

Getting more information on clinical trials, treatment, and care for GM1 gangliosidosis.

You have now found the Cure GM1 website on your internet search. We recommend you stop trying to sort through old papers and unfamiliar websites since so much of the information out there is out of date and confusing. The landscape is constantly evolving and changing. Much of the most current information is not published, many medical sites, even ones you deem trustworthy will give you an inaccurate and outdated information on what to expect.

Your doctors can assist you with selecting a clinical trial and contacting the trial’s principal investigators and sites. However, we recommend reaching out to us because we have the overall picture and we keep updates of all the press releases and results.
Please reach out for an informational letter and we will do our best to assist you in navigating the current landscape.

For more information, please contact info@curegm1.org

Resources for Newly Diagnosed

Here you’ll find useful information to start navigating GM1 with your loved one.

Your Story

It can be very tiring to keep repeating your story to doctors and to the general public.  It can be tiring to repeat the story to friends and family as well. Here are two ideas to help with the process of sharing your story.

  1. Create a “business” card which shares a bit about GM1 that you can keep in your wallet to share with people when you are out and about. This can keep you from having to keep repeating yourself and you can avoid stirring up emotions when you are not ready by offering the card instead of reliving your story constantly. We have created one for you – download it here.
  2. There is an app you can use to communicate with medical professionals, providers, and schools where you can story medical information and easily share it. This way, you are more organized and have a convenient way to share information and updates.  See: https://www.mymejo.com/
Get a Social Worker and Get Medicare

Most hospitals employ social workers. If no one offers to connect you in a doctors appointment, be sure to ask your doctors if there is one on staff. Also, be sure to ask for local resources such as a Regional Center.  

A social worker can help you apply for Medicare and other government services in your area. If the hospital does not have one on staff to help, you can ask if they know of contacts at the Regional Center or anyone else who can introduce you.

It may also be possible to apply for support through Social Security. It would be best to discuss with social workers and your community resources for more information in the ever-evolving landscape.

MEDICAID

Medicaid covers children with disabilities through state-specific waivers, offering insurance (usable as supplemental to eliminate copays) and respite care. Visit KidsWaivers or your state’s Medicaid agency for local programs. There’s also free or low-cost health and dental coverage through Medicaid & the Children’s Health Insurance Program (CHIP) – learn more on InsureKidsNow.Gov.

Get an Insurance Case Manager

When you are just starting out, there can be an overwhelming number of interactions with your health insurance company. It can be horrendous and time-consuming to sit in a phone maze all day trying to navigate your questions. Many insurance companies offer case managers. If you find yourself stuck in a phone maze all the time, be sure to ask your insurance company for a case manager. Get a direct phone line and email address with this person and make them work for you. It’s their job to help you.

Special Needs Planning Professional Guidance

There are services and professionals whose entire careers are to help people navigate “the system” and create a long term financial plan to your family. Cure GM1 has been partnering with All Needs Planning and Mary McDirmid. Their business is in Washington state, but they do help families with special needs planning throughout the entire United States. 

Counseling / Therapy

It can be helpful to seek private counseling or therapy. There are therapists that specifically focus on rare disease families. We can offer some names if you would like to explore this route.  Otherwise, your insurance company or workplace may be offer assistance in finding providers in your area which are covered by your insurance.

Give an Hour

Recognizing the unique struggles of rare disease caregivers, Give an Hour provides free mental health resources, peer support groups, and educational tools designed to empower caregivers in their mental health journeys. More >> https://giveanhour.org/supporting-rare-disease-caregivers

Financial assistance programs to help with medication, insurance premiums, co-pays, diagnostic testing, and travel to specialists

Charities

There are other charities that provide grants or services which can be helpful.

B Brave

B Brave Foundation provides customized grants tailored to each family and their individual journey. The funding is designed to ease financial stress on families and can cover a range of needs, including bills, medical expenses, lodging during hospital stays, funeral expenses and more.

More >> https://bbravefoundation.org/grant-application-form/

Chive Charities

Provides critical grants for individuals with life-altering or life-threatening needs. From therapy equipment like adaptive tricycles and robotic walkers to service dogs, wheelchair-accessible vans, and a wide range of mobility items, Chive Charities fills the gaps where insurance and other resources cannot.

More >> https://chivecharities.org

Hunter’s Hope

Hunter’s Hope Equipment and Supply Exchange program has been designed to help fulfill the equipment and supply needs of Leukodystrophy children as well as help families place equipment and supplies that they are no longer using. Hunter’s Hope will cover the shipping cost (48 contiguous states) to place the equipment with the family in need.

Equipment & Supply Exchange

Hunter’s Wish Gift helps families in the United States who face extreme financial stress attributed to the costs associated with caring for a child with Leukodystrophy. Requests are granted on a case-by-case basis and are limited to meeting needs that will enable an affected family to provide the best possible care for their child. (e.g. wheelchair lift, wheelchair-accessible van, service dog, etc.

Hunter’s Wish Gift
National Organization for Rare Diseases (NORD)

NORD provides patient assistance programs to help individuals living with rare diseases:

  • Obtain medication
  • Receive financial help with insurance premiums and co-pays
  • Get diagnostic testing assistance
  • Receive travel assistance for clinical trials or consultation with disease specialists
  • Provide caregiver respite
  • Offer support during emergencies
  • Gain knowledge about rare diseases
  • Connect with other patient assistance programs

More >> https://rarediseases.org/patient-assistance-programs

  • The Disability Resource Guide: Sierra Phillips, a rare disease mom, has created the ultimate resource guide for those diagnosed with or caring for someone with a rare disease, medical complexities, and disabilities. Download or read online: https://disabilityresourceguide.org
  • GM1 gangliosidosis disease and symptoms: Cure GM1 provides information on the disease here. In addition, here is the NIH’s informational page about the disease and latest findings.

Resources for our families

Take the next step in your GM1 journey by connecting, advocating, and making an impact.

  • NORD’s Rare Disease Educational Support Program: This program reimburses costs for rare disease-specific workshops, conferences, and educational programs. It can cover registration fees, travel costs, and lodging for attending educational events related to rare diseases, including nutrition workshops.
  • American Society for Gene & Cell Therapy (ASGCT): Offers patient-centered information and webinars on gene therapies
  • Genetics & Inheritance: This video explains the basics of genetics and inheritance, giving you examples of how conditions can be inherited and how DNA works.
  • Annual Cure GM1 Community Conference: Learn about current researches and connect with other caregivers: GM1 family registrations are free of charge.

There are many opportunities to support research and to participate in research such as providing bio-specimens, participating in registries, participating in focus groups, participating in natural history studies, and participating in clinical trials. The landscape is constantly changing and evolving. Cure GM1 works diligently to disseminate the most current information and opportunities.

Data Sharing
Participate in Research and Data Sharing

It is critical for an ultra-rare disease like GM1 to contribute to science and to help generate more data on the disease. We encourage families to keep abreast of all the requests to participate and to join in where possible. Depending on the languages and the government approvals and oversight, it may not be possible to participate.

Contact Us to Learn About Participating in Research

We recommend that you schedule a call with our team to review information. In addition, even if your doctors completed some outreach and tell you that there is nothing to be done, it can still be helpful to speak with us. It is important to also advocate for your own child directly. Too often, we have seen lost opportunities or families who have not been given current information.

Understanding Research Terms

We’ve created a glossary of common research terms to help you navigate scientific literature and clinical trial information

Learn more about the research landscape and Cure GM1 projects here.

  • The EveryLife Foundation is an American advocacy organization dedicated to empowering the rare disease patient community to advocate for impactful, science-driven legislation and policy.
  • The National Organization for Rare Diseases is a large umbrella U.S. organization supporting rare diseases and offer tools and guides on how to drive policy changes in your state or at federal level via advocacy.
  • Global Genes provides patient with services, trainings, Conferences to help you build your path to become a patient advocate through their Rare Advocacy Exchange Program.
  • Join our month-long awareness campaign leading up to GM1 Gangliosidosis Awareness Day on May 23rd. Each year, we collaborate with local and state governments to secure proclamations recognizing this important day. This initiative provides an excellent opportunity to educate policymakers while increasing visibility of the disease in your community. It helps us reach more healthcare providers, researchers, and families affected by GM1 Gangliosidosis.
  • There are many ways you can fundraise with us: here are some ideas.
  • Take action and contact us to brainstorm on ideas on how to participate. You can also engage in our fundraising campaigns which we present in our newsletters and on social media.
  • Storytelling is a critical component to advocacy. We must show that our families exist and the impact of GM1 on actual people’s lives. We offer many opportunities to share your story. Here’s a form to submit your story. A single post or photo can be a fundraiser.

Share your story

Personal stories create connections that facts alone cannot. When you share your GM1 journey, you transform a rare disease into a human experience that resonates. You can always submit stories to Cure GM1 to be shared. Sharing can help healthcare providers recognize symptoms in undiagnosed patients, inspire researchers, and show isolated families they’re not alone. If sharing feels intimidating, you can check free resources available for you:

  • Global Genes: gathers useful links and tips to help you share your rare story
  • The National Organization for Rare Diseases offers opportunities to share your story here.

Check here and get inspired by the stories told by our community.