rare disease
rare disease

Goodbye April & Hello May. 

Welcome to our special edition newsletter for rare disease International GM1 Awareness Day! As we approach May 23rd, we’re excited to unite globally to raise awareness for GM1 gangliosidosis, an initiative led by the Cure GM1 Foundation in collaboration with multiple advocacy groups. This year, gear up with our newly designed swag, available thanks to your votes, and show your support. We encourage everyone to get involved!

We’re also excited to announce upcoming events and achievements. Stay updated on news pertaining to GM1, rare diseases that could impact our community and prepare for summer with our fundraising initiatives and special holiday-themed gear for Mother’s Day. Your active participation fuels our journey towards a cure. Together, we can make a significant impact on the lives of those affected by GM1 gangliosidosis.

GM1 Awareness Day

Let’s unite globally for GM1 Day awareness on May 23rd. We hope to build a diverse international community and to unite as one to build momentum for our cause each and every year! Organized by the Cure GM1 Foundation in collaboration with multiple patient advocacy groups, the rare disease International GM1 Gangliosidosis Awareness Day, a worldwide movement.

Save the date, May 23rd! This day is an important moment for advocacy and awareness that will grow exponentially more powerful with each person who participates. Will you be one of those people and take part to help us spread awareness about GM1?

rare disease
rare disease

Ways To Support

We invite everyone, from patients, caregivers, families, clinicians, researchers, biotech representatives, policymakers, and anyone with an interest to participate in spreading awareness and taking action to help those affected by a rare disease GM1 gangliosidosis. Collectively, we can make big change. Three easy was to support the fight against GM1. First is to donate, your donations fuel research, advocacy, and awareness. Second, write your representative or government officials to have International GM1 Gangliosidosis Awareness Day proclaimed as an official day of observance Learn More. Third, is to buy awareness gear from our store to support the cause in style. Wear it proudly and join the GM1 fight. Order Today.

Gear Up

GM1 Awareness Day is fast approaching, and thanks to your votes, we’ve selected two top designs for our GM1 Day swag! Make sure to place your orders in time to showcase your support by May, 23, 2024.

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Cure GM1 Virtual Annual Conference

2024 Virtual Annual Conference

Please save the date and prepare to join us on September 20, 2024, the conference will be held virtually. This significant event is your gateway to the latest in research, trials, and more. Dive into the latest rare disease GM1 research and community stories at our 2024 virtual conference. Join us from the comfort of your home on September 20th. Stay tuned for more details!

Spotlight on Advocacy!

We are thrilled to announce that our founder and president, Christine Waggoner, will be representing our advocacy efforts at the upcoming Seattle Rare Disease Fair. Join her on May 31st to dive into the strides we’re making in the fight against GM1 gangliosidosis. If you’re in the Seattle area, don’t miss this opportunity to connect with the community at the Seattle Children’s Research Institute from May 30th to June 1st. This event is a beacon for anyone touched by rare diseases and a chance to come together to learn, share, and support one another. See you there!

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Let’s Get Started- Rare Patient Voice 


Are you a patient or family caregiver with something to say? Make your voice heard by participating in paid surveys, interviews and online communities! Start talking to the right people. It’s free! We accept rare and non-rare diagnoses! In celebration of our 10th Anniversary our studies now pay at a rate of $120 an hour!

Celebrating Achievements in Research

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Cure GM1 BioTech News

Sanofi Press Release: AMETHIST Venglustat Trial Discontinued

It is with heavy hearts that we share the news of the discontinuation of the AMETHIST trial for venglustat on the 25th, a moment that has deeply impacted many families within our community. This trial, which included a basket trial arm for GM1 gangliosidosis patients, was a beacon of hope for many. Unfortunately, this marks the fourth closure of a GM1 study in recent years, following a promising surge of trials beginning in 2019. Despite these setbacks, including bankruptcies and discontinued projects, our resolve remains unshaken.

The journey toward finding effective treatments for GM1 illustrates that overcoming this challenge transcends scientific inquiry alone. It demands meaningful patient engagement and robust collaboration, ensuring that trials are designed to truly benefit those they intend to serve. Moreover, the need for improved regulatory pathways and a modernized newborn screening system is more apparent than ever, as these are crucial for diagnosing and qualifying trial candidates early.

At the Cure GM1 Foundation, we truly understand the heartbreak of GM1 and the power and importance of patient advocacy. We continue to advocate, innovate, and push forward, knowing that our collective efforts are vital. The path to finding a treatment is fraught with challenges, but together,  we must continue and unite towards achieving our goals. The full press release is available here: Read More.

Differences Between A Gene Therapy Clinical Trial And A Cure

“A clinical trial is a research study required by the FDA before a drug is available on the public market. The purpose is to test the safety and ability of a new therapy to improve a specific disease state or “indication”. Based on clinical trial data, the FDA decides if a drug is approved or not. To say that an FDA-approved drug works and a rejected drug does not work, however would be limiting.

Clinical trials, especially in the case of rare diseases and gene therapy, are not so black and white. In rare disease clinical trials with patients that have varied experiences with the same condition, it can be difficult to measure whether a treatment is effective. This article will explain how clinical trials are set up, the unique features of testing new treatments in rare conditions, potential differences in receiving gene therapy post-approval, and set expectations around the potential of gene therapy.” Read More

Over 1 in 3 people Affected by Neurological Conditions

“Neurological conditions cause great suffering to the individuals and families they affect, and rob communities and economies of human capital,” said Dr Tedros Adhanom Ghebreyesus, WHO Director-General. “This study should serve as an urgent call to action to scale up targeted interventions to allow the growing number of people living with neurological conditions to access the quality care, treatment and rehabilitation they need. It is more important than ever to ensure brain health is better understood, valued and protected, from early childhood to later life.” Read More

Study Lists Neurological Disease as the Top Cause of Disability and Illness Around the World

“Although more than 80% of the neurological deaths and nervous system disorders occur in lower income countries, people living in high-income countries have about seventy times the number of neurological professionals per 100,000 people. Dr. Tedros Ghebreyesus, Director-General of WHO said that the study is an urgent message that brings awareness of the increasing number of individuals who are in need of treatment and rehabilitation. Dr. Ghebreyesus emphasizes the need for an improved understanding of brain health starting in early childhood.” Read More

Coping Skills for Caregivers Webinar

Featuring Suzanne Edison, Mental Health Coordinator & International Family Support, from CureJM Foundation, we will be hosting our second caregivers webinar May 9th, 3 pm – 4 pm ET. Learn about the factors affecting mental/emotional health, the social and physical changes that kids experience that lead to differences from their peers, increased stress, anxiety, and possible depression.

Celebrate All Super GM1 Moms This Mother’s Day

With Mother’s Day fast approaching, it’s the perfect time to honor the remarkable moms in our Cure GM1 community. We’d love to shine some spot light & recognition to our Super Moms out there, so if you have someone in mind we would be thrilled to hear from you. Please reach out to anita@curegm1.org to share their story. Let’s celebrate these incredible women together!

This Mother’s Day, show the incredible moms in your life how much they mean to you with our exclusive “SUPER GM1 MOM” shirt! It’s the perfect way to honor her strength, love, and unwavering support. Wish her the best day ever and let her wear her superhero status proudly. Order now and make this Mother’s Day one to remember. Your purchase supports the vital work of Cure GM1, making this gift truly meaningful.

Ways to Give – Your Support Matters

RaiseRight | Charity Miles | Facebook Fundraisers | Donate 

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