Goodbye May & Hello June.
The CURE Gm1 Catalyst
In this month’s newsletter, we have a range of important updates and heartwarming stories to share. Registration is now open for Cure GM1’s 2024 International GM1 Virtual Community Conference. We celebrate proclamations, highlighting all the support we received on GM1 Day.
Do not miss our updates on Cure GM1 community engagement, including the success of Armand’s Prom and Christine Waggoner’s impactful speech to the Federal Rare Disease Caucus and her attendance at a special scientific workshop in Washington, DC, are also covered. We wrap up with our fundraising efforts, featuring Father’s Day t-shirt designs and a celebration of birthdays and angelversaries. Join us as we continue to raise awareness and support for our community.
INTERNATIONAL GM1 VIRTUAL CONFERENCE
INTERNATIONAL GM1 AWARENESS DAY RECAP
COMMUNITY ENGAGEMENT
FUNDRAISING
ANGELVERSARY & BIRTHDAYS
Cure GM1 Annual Conference: Registration is Now Open!
INTERNATIONAL GM1 VIRTUAL CONFERENCE
We are excited to announce that registration is now open for the Cure GM1 Foundation’s Annual International GM1 Virtual Community Conference, set to take place on September 20th. This conference is an invaluable resource for families, researchers, and biotech companies, bringing together a global community dedicated to finding treatments for GM1 gangliosidosis.
Thanks to the generous support of our event sponsors, families can attend this virtual conference free of charge. Each year, the GM1 Virtual Community Conference fosters a sense of solidarity and shared purpose, serving as a platform where real change begins. Through advocacy, increased awareness, and the acceleration of drug development, this event galvanizes our community and drives forward our mission. Join us on September 20th and be a part of this transformative event.
thank you for Helping Raise GM1 Awareness on May 23rd.
The goal of this day was to bring recognition for GM1 gangliosidosis to broaden awareness of this devastating and fatal disease.
Wisconsin Governor Tony Evers declared GM1 Day
INTERNATIONAL GM1 AWARENESS DAY RECAP
On May 23rd, Wisconsin Governor Tony Evers declared Gangliosidosis Awareness Day, highlighting the urgent need for increased awareness and support for those affected by these rare genetic disorders, including GM1 gangliosidosis. Lindsay’s proclamation, featured in a WEAU news segment, emphasizes the importance of research funding and improved diagnostic tools to enhance patient outcomes. Governor Evers’ proclamation is a significant step towards greater recognition and understanding, aiming to inspire community support and drive efforts towards finding a cure. Watch full segment.
2024 Proclamations
INTERNATIONAL GM1 AWARENESS DAY RECAP
This year, we are excited to share the many proclamations we have received, each one serving as a testament to the growing awareness and support for GM1. These proclamations are a powerful acknowledgment of our collective efforts and the importance of our cause. We extend a heartfelt thank you to everyone who took the time to complete each step in this process. Every action, no matter how small, contributes significantly to our mission. For those who missed the cutoff this year, don’t worry—there is always next year. Your continued support and involvement are crucial, and we look forward to your participation in future efforts. Together, we can keep pushing forward, making every step count, and ensuring that our voices are heard.
Celebrating Armand: A Memorable Prom Night
COMMUNITY ENGAGEMENT
We are delighted to spotlight Armand, who recently enjoyed a memorable prom night. This heartwarming event, covered by WNEM, highlights the incredible support and love from his community. Armand’s special night is a testament to the power of coming together to create joy and lasting memories for those affected by GM1. At Cure GM1 Foundation, we celebrate moments like these that bring happiness and hope to families navigating difficult journeys. Watch full segment.
Rare Across America Registration Is Now Open
COMMUNITY ENGAGEMENT
Rare Across America 2024 is the perfect opportunity to meet with your Members of Congress at their in-district offices. You can educate them on the issues that are most important to the rare community by sharing your story.
Christine Waggoner Speaks at Federal Rare Disease Caucus, Advocates for Ultra-Rare Disease Treatment Development
COMMUNITY ENGAGEMENT
Christine Waggoner had the honor of speaking to the federal rare disease caucus on May 20th to help share the experiences and challenges of the development of possible treatments for ultra-rare diseases. Information on the briefing session Read More.
At the workshop entitled “Novel Approaches to Drug Development for Small Rare Disease Populations—and the Policies that Can Drive Them” in DC, it was a pleasure to meet up with Aidan’s family at the congressional briefing where little Aidan who suffers from late infantile GM1 had the opportunity to meet with congressional staff members. Christine Waggoner had the opportunity to attend a special scientific workshop in Washington DC. See this like for title and more information: Read More.
Federal agency and patient advocacy community leaders, regulatory experts and industry professionals and others will come together to inform the ongoing debate regarding the necessity and potential framework of a formal definition for ultra-rare diseases and the policies that are needed to drive successful therapy development. Recordings may be available through EveryLife: https://bit.ly/3crHRGd
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.
Championing the Cause: Christine Waggoner’s Fight for GM1 Gangliosidosis Treatments
COMMUNITY ENGAGEMENT
On May 31st, our President and Founder, Christine Waggoner, took to the stage to deliver a powerful talk titled “Fight for Treatments for GM1 Gangliosidosis.” Her presentation, given at Seattle Children’s Hospital at the NW Rare Disease Fair in Washington, aimed to shine a spotlight on the urgent need for treatments and raise awareness about GM1 gangliosidosis, a rare and devastating genetic disorder.
Christine’s journey with GM1 gangliosidosis began when her own daughter Iris was diagnosed with this heartbreaking disease. Since then, she has dedicated her life to advocating for treatments and ultimately a cure. Her passion and determination have been the driving force behind the Cure GM1 Foundation, an organization that has made significant strides in research and patient support.
During her talk, Christine highlighted the challenges faced by families affected by GM1 gangliosidosis. She spoke about the relentless progression of the disease, which gradually destroys nerve cells in the brain and spinal cord, leading to severe neurological impairment and a drastically shortened lifespan. Her heartfelt stories of affected children and their families resonated deeply with the audience, painting a vivid picture of the urgent need for medical advancements.
Christine also emphasized the importance of community and collaboration in the fight against GM1 gangliosidosis. She called on researchers, healthcare professionals, and the rare disease community to join forces in accelerating the development of effective treatments. Her message was clear: together, we can make a difference and bring hope to those living with this devastating condition.
The NW Rare Disease Fair provided an excellent platform for Christine to raise awareness about GM1 gangliosidosis. The event, which brought together patients, families, researchers, and advocates from across the rare disease community, served as a powerful reminder of the strength and resilience of those affected by rare conditions. Christine’s talk was met with overwhelming support and appreciation, further fueling her determination to continue the fight for a cure.
As we reflect on Christine’s impactful presentation, we are reminded of the critical role that awareness and advocacy play in driving progress. Every speech, every conversation, and every shared story brings us one step closer to finding effective treatments for GM1 gangliosidosis.
Plan a Summer Fundraiser
FUNDRAISING
Did you know you can fundraise on behalf of Cure GM1? Individual fundraisers are one of the most powerful ways to support Cure GM1. They can be done online or in person, can be simple or ambitious, and can occur over a long period of time or in one day.
Father’s Day
FUNDRAISING
Celebrate Father’s Day with our special GM1 T-Shirts! Perfect gifts to honor super dads. Order now!
Become A Sponsor for 2024 annual conference
FUNDRAISING
Sponsorship covers overhead costs and keeps the conference free for families. Become a sponsor of the 2024 conference today to support patient advocacy, awareness, and community. Contact us now at info@curegm1.org
