Move for GM1!

Today is the kickoff for the Move for GM1 summer fundraiser! Join us throughout August and September. It’s so easy to participate: 

  1. Download the Charity Miles App
  2. Log miles for Cure GM1 via your activity of choice
  3. Share your progress on your social profiles after every exercise session
  4. Get donations from some of the people who see your post

For all those who care about making progress toward effective, approved treatments for GM1, this is one of the most easy and effective ways to make a difference! Don’t miss this opportunity to earn up to $10K in matching funds, and get moving for GM1!  

The Conference is Fast Approaching

The 2023 GM1 Conference will be here before we know it! Make sure to register today to reserve your spot. Attendees can join this hybrid event either in-person or virtually, though there are a lot of perks to coming in person, including FREE tickets to the Oakland A’s game on Saturday the 23rd following the conference. Don’t miss out!

Azafaros is also organizing a focus group to learn more about GM1 the morning after the conference. If you have already registered, please let us know if you are available and willing to participate.

News

Congratulations to Christine! 

Cure GM1 founder Christine Waggoner was included in Endpoints’ list of 20 women leaders shaping biopharma research and development in 2023! They’ve discovered what most in our community have known for a long time, that Christine is a relentless advocate for GM1 and that we would not have made the progress we have without her. We are truly grateful for the honor, recognition, and greater awareness for GM1 and rare disease moms. Thank you Endpoints News for enabling our work!

Contribute Samples to Biorepository and Biomarker Study

Cure GM1 is collaborating with both Combined Brain and the Gelb Laboratory to organize a biomarker study to help improve newborn screening diagnostic testing. Families may participate from the comfort of their homes to provide blood and urine samples. Please mail info@curegm1.org if you are interested in participating.

Combined Brain is a nonprofit working to speed the path to clinical treatments for people with rare genetic neurodevelopmental disorders by pooling efforts, studies, and data. We are grateful for their partnership in helping support biobanking at Vanderbilt and the Gelb study.

Community

New Study on GM1 in Adults

Dr. Sylvia Stockler, biochemical geneticist at BC Children’s Hospital, is starting a study on adults with GM1 to study patient reported outcomes and the development of outcome measures for adults with GM1. An adult with GM1 could be either Type 2b or Type 3. For example, there are adults with GM1 juvenile / 2b who are over 18. If you are interested in helping or participating, please consider joining the call with Dr. Stockler. The call will take place on Monday, August 28th at 8am PT. Please email info@curegm1.org to reserve your spot.

Gene Therapy Caregiver Experience Focus Group

If you or your GM1 loved one has participated in or is considering participating in a pediatric GM1 gene therapy clinical trial, and you live in the US, you may be eligible to join a paid focus group. NYU is recruiting for a study about the caregiver experience with pediatric gene therapy. The purpose of this study is to gain more insight into the lived experience (i.e., emotional, social, ethical, and equity trade-offs that impact lives and illness experience) of pediatric gene therapy. You will be compensated for your time. If you are interested, please email Cara Hunt at Cara.hunt@nyulangone.org.

Nominate your GM1 Loved One for Go Shout Love 

Go Shout Love is an organization that strives to have a positive impact on families with kids on rare medical journeys by raising awareness, funds, and creating community through social media. Every month they select a different family from a pool of nominees, then design inspirational, unique, and positive apparel inspired by the featured child. They spend all month celebrating that family and shouting love for them. Funds raised by the sale of their apparel go directly to the featured family to help with items related to caring for their child.

There are many amazing GM1 families who would be wonderful nominees. Nominate your GM1 loved one today.

National Immunization Awareness Month

August is National Immunization Awareness Month. Vaccination is critical to the health of people of all ages, but especially to those who are immunocompromised, like many in the GM1 community. To help spread awareness you can visit the National Immunization Awareness site for a wealth of information and resources.

Happy Birthday

  • Mauro – July 25th
  • Aryana – July 25th
  • Cristal – July 25th
  • Daxton – July 28th

In Loving Memory

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