Ways You Can Help Cure GM1

1. Donate

The single most effective way you can help Cure GM1 is to donate money. Nearly all the money we raise goes into research for treatments and a cure for GM1 Gangliosidosis. Most everything else you can do would facilitate spreading awareness to generate donations. At the end of the day, money is what drives research–and what limits it.

If you want to donate in a way that doesn’t involve direct payments, you can. We accept donations of stock and used vehicles, which don’t have to be running. Please also take advantage of your corporate matching program, if your employer has one.

2. Fundraise for Cure GM1

When members of our community host fundraisers, you expand our audience to your network. This is very powerful for a few reasons. First, it spreads information about our organization to people who wouldn’t otherwise see it. Second, the people you know trust you much more than they trust us, an unknown organization. They are much more likely to donate when you ask than when we do. And as you know, donations are critical.

Remember, there is no shame or harm in promoting our cause. We are trying to save the lives of those who suffer from GM1.

There are many opportunities and methods to fundraise throughout the year. Take a look at our Fundraise for GM1 Page and our Fundraising Manual for more information.

3. Engage on Social Media

Social media is a critical tool we can use to bring awareness to GM1 and our organization. We are seeking help with tasks such as sharing the stories of those newly diagnosed, responding to community posts, creating new content, and supporting fundraising campaigns.

4. Advocate for Better Legislation

Often times laws are proposed that would positively or negatively affect the quality of life for GM1 patients, as well as the ease of access to essential medications, therapies, and equipment they rely on. Contacting your lawmakers can affect these outcomes.

Good advocacy resources include:

5. Join a Clinical Trial

Clinical trials are research studies which evaluate the safety and efficacy of a new medical intervention in people. In the case of GM1 Gangliosidosis, there are multiple AAV gene therapy trials in progress. 

You can also help support research by supporting studies that will help deepen the understanding of GM1 such as participating in interviews, focus groups, and pilot studies. In these activities, there is no treatment.There are several clinical trials currently recruiting and testing treatment for GM1 Gangliosidosis.

The more people that participate in these trials, the faster that treatments may become available for GM1 patients. By participating in a clinical trial, you help everyone suffering from GM1. Contact us to ask us about the overall trial landscape.

6. Participate in Data Collection

Cure GM1 partners with two initiatives which collect patient data: Invitae Patient Insights Network (PIN) and AllStripes. Both initiatives collect data, both strive to characterize the patient journey and the patient community, and for both, patients own their data.

The purpose of these initiatives is to accumulate data from as many patients as possible, and use this data to inform research and advance the knowledge of GM1 so doctors can give better care. In the case of Invitae PIN, patients will also be able to better understand their own health and condition as well as learn about research and clinical trials.

  1. Invitae PIN – This is a patient centered registry where patients can share their de-identified health data and experiences. This is the largest GM1 patient registry of any patient advocacy group. They provide insights based on patient-reported outcomes and gather genetic reports in collaboration with Genome Connect.
  2. AllStripes – This is a rare disease data analysis company. AllStripes provides data insights through the analysis of medical records to aid researchers and drug developers in creating and accelerating treatments for GM1.

If you are the caregiver for someone with GM1, please consider participating in data collection. The more data that becomes available, the faster treatment can be developed. If you participate in one or both of these data collection initiatives, you will help our cause enormously.

7. Take Part in a Natural History Study

A natural history study is one that follows a group of people with a specific disease, which has no intervention. The study takes place over a long period of time, from the onset of the disease over the course of the patient’s lifetime. The information gathered can include patient symptoms, testing samples, the care they receive, clinician reports, and quality of life evaluations. Researchers study this data to better understand the natural progression of the disease, the results of which can then serve as a comparator to see whether or not a treatment is working.

In the case of rare diseases, like GM1 Gangliosidosis, this information is critical in the development of drugs. Unfortunately this information is usually not available to researchers or is incomplete. This makes the data that does get collected especially valuable. It has the potential to bring about new understandings and breakthroughs, and can be used to develop safe and effective drugs and treatments. 

When you participate in a natural history study, you are helping everyone who suffers from GM1. Take a look at the natural history studies currently recruiting.

8. Sign a Media Waiver & Share Your Story

Sign our media waiver and send us photos of your family that we can use in our media and outreach. Photos are very persuasive and humanize this disease in a way that facts and research can’t. When you allow us to use your photos, we can spread awareness much more effectively.

Share your story so we can let those less familiar with GM1 understand what a journey to diagnosis can look like and what it’s like to manage this disease day to day. The more people know, the more they’ll want to help us create change.

9. Subscribe to the Cure GM1 Catalyst

By signing up for our monthly newsletter you’ll get updates about GM1 research initiatives, information about our most recent awareness campaigns, and news about the GM1 landscape and community. It’s a great way to stay continually active and informed.

10. Attend our Annual Conference

Most every year Cure GM1 hosts a conference where clinicians, researchers, biotech representatives, and patients meet to learn about clinical trials and research progress, to hear perspectives from doctors and patients, and to meet and connect with one another. Attending strengthens our community. It also helps us spread awareness, share important information, and gives you a space to receive support and community from people who share your experiences. Registration becomes available a few months before the conferences, which we’ll announce well in advance, so follow us on social media and subscribe to our newsletter.

11. Support Us While You Shop

Many retailers offer options to select a charity as your default so that a portion of your purchase gets donated to us. These include:

You can also support us through regular fundraisers from vendors who have charitable programs. Proceeds from fundraisers through these partners help support research:

Every Little Bit Counts

No matter how you choose to support us, it helps and it matters. We appreciate the time and effort you take to help us. Because of you, we get closer to finding treatment for GM1 Gangliosidosis every day. Thank you.

Ways to Give – Your Support Matters

Charity Miles | RoundUp App | Walmart RoundUP | Facebook Fundraisers | Donate | Set up a Recurring Donation

Visit our Take Action page for more ways to support our community