Cure GM1 Catalyst

COMMUNITY ENGAGEMENT
ADVOCACY
BIOTECH NEWS
FUNDRAISERS
ANGELVERSARY & BIRTHDAYS

A note from Christine

Dear GM1 Community,

This month brought exciting advances with the announcement of the first successful personalized CRISPR gene editing treatment for a baby with a rare disease. Many have asked about the implications for GM1. This personalized treatment developed for CPS1 deficiency addresses a different condition than GM1. GM1 affects the complex GLB1 gene that produces the beta-galactosidase enzyme throughout the brain, spinal cord, and body. GM1 has significant neurological symptoms and is a degenerative brain disease. The treatment approach for CPS1 was liver-directed. We continue to closely monitor all therapeutic approaches and their potential applications to GM1. We congratulate all those involved on this significant milestone in gene editing. We remain hopeful for the continued progress of the therapies currently being developed for GM1 that can serve a broad group. 

We would like to thank everyone who participated in making GM1 Day a tremendous success. Your participation was inspiring and incredibly important. We secured proclamations throughout the country and reached the news. We must continue to show that we exist and our families and children deserve a brighter future.

We’re proud to announce that Cure GM1 Foundation has received the Gold Candid Seal, recognizing our commitment to transparency and accountability. This honor reflects our dedication to responsible stewardship of your generous donations as we pursue treatments for GM1.

Finally, check our new Family Resources Page on our website. We’ve created a comprehensive hub gathering useful information and tools for families in one location. Let us know if you have any feedback!

Highlights from this Month
  • Gold Candid Seal achieved
  • New Family Resources Page on our website
  • 12 proclamations secured for GM1 Day
  • Azafaros announces fund to their phase 3 clinical trials for nizubaglustat
  • Conference registration is open! Travel scholarships and Disneyland passes are still available. Rules and limitations apply.

Community Engagement

GM1 Day Highlights

Your engagement in our GM1 Day Campaign during May blew our minds! The GM1 community showed incredible strength and unity during GM1 Awareness Month.

We’ve secured 12 proclamations recognizing GM1 Day, 5 of them for the first time, a record number that increases our visibility and support. The proclamations establish GM1 as a recognized cause worthy of attention and resources and brings significant visibility to our cause.

States: California, Illinois, Maryland, Massachusetts, Michigan, Missouri, Texas, Wisconsin, North Carolina; Cities: Carlsbad (CA), Milwaukee (WI), and Village of Rantoul (IL).

David Law and Christine Waggoner attended the plenary session at Sacramento in which Senator Padilla proclaimed May 23rd as GM1 Awareness Day in California. Watch the Session Here

Ashley and Connor bravely shared Ezra’s GM1 Story with their local media. Watch the heartfelt interview here

Your social media posts, photos, and heartfelt stories spread awareness far beyond our immediate circle. We extend our deepest gratitude to everyone who made #GM1Day campaign a success!

Father’s Day Tribute – Deadline Extended to June 10!

Let’s honor the unique fathers and father figures in our GM1 community! Send a photo and brief quote about what makes them special to tayara@curegm1.org by June 10th. Let’s celebrate these heroes who provide strength, support, and love throughout our GM1 journeys!

Dominic’s Experience with Intravenous AAV9 Gene Therapy

Dominic is a playful two-year-old who has been diagnosed with GM1 Type 2b and recently enrolled in the clinical trials for intravenous AAV9 gene therapy at the NIH. He is the third of three brothers to join NHGRI studies on GM1 gangliosidosis. Read about how Dominic and his brother Oliver’s GM1 gene therapy treatment has progressed here.

Fionas’s Story

This June, we remember Fiona’s birthday and honor this brave little girl who taught lessons of love, joy, and living in the moment. Her story reminds us why our work matters and shows the incredible strength of families facing rare diseases. Her legacy teaches us what truly matters: family, kindness, love, and hope. Read here her father’s beautiful and moving account of their journey—from diagnosis to the devastating progression of GM1.

Connect and Join Us at the Annual Cure GM1 Conference!

Registration for our annual Conference is open! Join us August 7-8 in Irvine, CA, for a day of research updates, family networking, and expert panels. Families attend free of cost. Disneyland passes and travel scholarships are still available on a first come first serve pass. Rules and limitations apply. Register Here

We would love to see you in person!  However, we do recognize how difficult it can be to travel for long distances and do have virtual ticket options.
There are many ways to support the conference:

Advocacy

David Law Featured in The Portrait Project

We’re proud to share that our community member David Law is now featured in The Portrait Project by Mural Health. This initiative humanizes rare diseases through personal stories, helping the public and medical community better understand the human impact of conditions like GM1. David’s inclusion helps represent GM1 in this advocacy work, increasing visibility for our cause and connecting our community to the broader rare disease movement. Read his and Violet’s story

Cure GM1 Signs Letter Supporting Accelerated Approval Pathway

Cure GM1 Foundation has joined other rare disease organizations in signing a letter urging the FDA to continue using the accelerated approval pathway. For rare diseases like GM1, where patient populations are small and disease progression can be devastating, accelerated approval represents a vital path to getting treatments to patients more quickly – without lowering standards. Read the letter here

NHC Innovation Symposium

Our founder Christine Waggoner represented Cure GM1 at the National Health Council’s Symposium on medical innovation. The event gathered leading healthcare experts to discuss rapid advancements in medicine, medical technology, and artificial intelligence, providing valuable insights and networking opportunities for our advocacy work. Learn more about the event

Biotech News

Azafaros Secures €132M for Lysosomal Storage Disorder Therapies

Azafaros has secured investment to advance Phase 3 clinical programs for nizubaglustat, for both GM1/GM2 gangliosidoses and Niemann-Pick disease Type C (NPC) later this year. The drug has a unique dual mode of action and is orally available and brain penetrant, designed specifically for rare lysosomal storage disorders with neurological involvement. This investment will accelerate development of the drug, which has already received Designations for Rare Pediatric Disease, Orphan Drug, and Fast Track from the FDA. Read full press release here

First Personalized Gene Editing Success

KJ, a 9-month-old with a rare condition, has been successfully treated with the world’s first personalized gene editing treatment. This breakthrough resulted from unprecedented collaboration between researchers, industry partners, and government agencies who created this treatment in just six months. NIH funding was instrumental, demonstrating how targeted resources can accelerate treatments for rare diseases when commercial incentives are lacking. Read the full story here

Fundraisers

Ben’s GM1 Day Fundraisers

During GM1 Awareness Month, Ben’s family organized multiple fundraisers throughout the month. They distributed flyers, organized events, and solicited support from local businesses.

Spring and Summer Fundraisers

Spring and summer are excellent times to fundraise. The weather is great for a walk or lemonade stand or for whatever creative activity you choose. This is a great time to Join Charity Miles and ask your friends to sponsor you for walking, running, or biking.

Host your own!

Want to host your own fundraiser? Visit our Fundraising Ideas page and check our guide to help you organize a fundraising.

Learn all about fundraising for Cure GM1

Angelversaries and Birthdays

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.

Cure GM1 Foundation | PO Box 6890 | Albany, CA 94706 US