Thank You Donors

We raised $76K at the end of 2023 thanks to our generous supporters and community. Thank you to everyone who donated. We are thrilled to be able to continue this work in 2024 and are dedicated to advancing research and policy so that possible future treatments for GM1 will ultimately become available. 

Beyond gifts, if you have supported Cure GM1 in any way – be it through fundraising, advocacy, or participating in our community – your efforts have advanced our cause. Thank you for your participation, your support, and your belief in us. We couldn’t do this without you.

2023 Impact Report

We are incredibly proud of all we did in 2023. Among our accomplishments are research studies and preclinical research projects, as well as truly meaningful advocacy and community-building efforts. Many GM1 families and advocates put on more than a handful of notable fundraisers. To see the complete list, take a look at our 2023 Impact Report.

Wish Upon a Star

Many people sent in beautiful wishes for the future of GM1. Thank you everyone who participated. It gives us hope to see what promising outcomes look like for others. You can read the wishes here.

Research News

We are sad to share that the Passage Bio Imagine-1 gene therapy study for GM1 has been paused. As the third GM1 gene therapy halted, this is devastating news. We know it’s easy to feel defeated and pessimistic about this, but not all hope is lost. 

The Imagine-1 program is only paused, not canceled. The press release mentioned they are looking for a partner to resume the program in the future. GM1 is also included in small molecule clinical trials, which are steadily progressing. Azafaros recently shared a press release on completing enrollment for their Phase II study. You can read that here.

Cure GM1 has several projects underway that have yet to be announced due to the contracting process. Rest assured that behind the scenes, we are busy and we will share and announce these efforts as soon as possible.

Cure GM1 is committed to taking on the challenges of advocating and funding new research in 2024 and beyond. We care about this disease regardless of setbacks and will keep working toward change.

Provide Samples for CombinedBrain 

CombinedBrain is Cure GM1’s new GM1 Biobank. They are in the process of collecting and storing blood, tissue, and urine samples from GM1 patients. These samples are critical for identifying new biomarkers, which will help improve GM1 research and diagnosis. The more samples we have, the better.

If you have the capacity, we urge you to contribute. Providing samples is free and simple. GM1 patients and families may participate from the comfort of their homes. Please mail info@curegm1.org to get started.

Advocate for Quality of Life

January is International Quality of Life Month. Help us humanize this disease and spread awareness about the importance of improving quality of life for GM1 patients by sharing your story. You can do this by submitting a share your story form on our website, or by reaching out to your local press.

Coming Up in 2024

Rare Caregivers Peer-Support Training

Give an Hour is hosting a free Rare Caregivers Peer-Support Training on January 29th at 5:00 PM – 8:00 PM EST, where you will have the chance to learn from and connect with others who understand the unique challenges you face. All GM1 caregivers are welcome to attend. Register here.

WORLD Symposium 2024

WORLDSymposium is a conference that features platform and poster presentations on research and advances in lysosomal diseases. This year it will take place from February 4th – 9th in San Diego, California. Registration is FREE for GM1 families. If you can’t attend, registration will allow you to watch recordings of the presentations. Cure GM1 founder and president, Christine Waggoner, will be in attendance and would love to meet you.

What are your 2024 Resolutions?

We’ve added these two GM1-related resolutions to our list. If you’re looking for some good ones, we don’t mind if you copy us!

  • Self-care, especially for caregivers. We plan to make sure we fill our cups – we won’t be able to give to others if we’re running on empty. We plan to take advantage of therapy, mindfulness, walks, baths, journaling, alone time, hobbies, exercise, and more. 
  • Making a difference – When we feel overwhelmed or hopeless about GM1 or our loved ones, taking action gives us a much-needed sense of control and empowerment. Simple ways to make a difference include putting on an online fundraiser for a birthday or special occasion, participating in important advocacy events, volunteering, telling our stories, and engaging with the GM1 community online.

Opportunities to take part in 2024 include: 

  • Rare Disease Day – February (Rare Disease Week is February 25th – 28th)
  • GM1 Awareness Day – May 23rd
  • Move for GM1 – August 1st – September 30th
  • Virtual Fall Conference – Fall
  • Giving Tuesday – Dec 3rd

Ways to Support Cure GM1 in January

In Loving Memory

Ways to Give – Your Support Matters

RaiseRight | iGive | Charity Miles | Facebook Fundraisers | Donate 

 Set up a Recurring Donation

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