WHAT WE DO
We are changing the Status of GM1
GM1 gangliosidosis was first identified as a distinct disease in 1968 and yet, patient advocacy and a strong focus on GM1 was sorely lacking until recently. Cure GM1’s focus is always on GM1. The Cure GM1 Foundation’s mission is to fund research and to support patient advocacy and drug development for the benefit of all those who suffer from GM1. This 501(c)(3) nonprofit organization was founded by parents of children who suffer from GM1. The U.S. federal government approved the formation of our organization in April of 2015.
~$5.4M
Cure GM1 has raised significant funding and driven meaningful progress, but more funding is still required.
4
Since Cure GM1 was founded, there have been 4 clinical trials, whereas prior to Cure GM1’s existence, there were none.
95%
95% of the 10,000+ rare diseases have no treatments.
Community Stories
Special Events
Participate and Take Action
International GM1 Community Conference
Our conference is an extremely valuable resource to GM1 families and to researchers and biotechs working on GM1. The presentations provide the most current information regarding the overall landscape.
Move for GM1
Move for GM1 is a simple fundraiser that takes place in August. Those who participate log miles walking, running, biking–or anything else–using the Charity Miles App, then share their progress on social channels so others can donate.
Rare Disease Day
Raising awareness and generating change for the 300 million people worldwide living with a rare disease, their families and caregivers.
International GM1 Gangliosidosis Awareness Day
Organized by Cure GM1 in collaboration with multiple patient advocacy groups, International GM1 Gangliosidosis Awareness Day is a worldwide movement. Increased advocacy and the funding of research and drug development can help advance the creation of possible treatments and ultimately improve and save lives.
Take Meaningful Action and Help Advance our Mission
Our work depends on volunteers and active participation in GM1 research, advocacy, and fundraising. Watch a video about our critically important work.
Helpful resources
Help Build Resources, Tools, knowledge and Community
Donations, volunteers, and our global community fuel our critically important work to help develop and advance possible treatments.
GM1 and rare disease community
collaboration, advocacy and community are critical
Cure GM1 has been instrumental in helping build community and to structure meaningful projects and collaboration amongst stakeholders. Some highlights include the organization of a natural history data sharing effort, the development of a newborn screening assay, and interactions with the U.S. Food and Drug Adminstration, FDA.
Jenny Bragg,
GM1 parent
“If I could change Clara’s future, I would. Nevertheless, I am forever grateful for the impact she has had on my life as well as countless others.”
Kylie Harrison,
Board Member
“People are telling us there’s nothing we can do for our daughters, we’re not accepting that. There is something we can do and that is to raise awareness and raise funding for them and for others.
Christine Waggoner
Founder
“Cure GM1 is laser-focused on creating a legacy of hope and change by navigating the many challenges of ultra-rare disease drug development and research.”
Emil Kakkis, Advisor
“I believe that GM1 is at a place where it can get treated. But you have to be vigilant and fight for support to ensure that the right things get done and that treatments become available.”
SAFE + EASY DONATIONS and fundraising
Ways to Donate and Fundraise
Download our fundraising manual. Read our refund policy.
Contact us for help
Email info@curegm1.org
Board Members, Staff, Volunteers
A Passionate and Experienced Team Fueling Our Work
Staff
Christine Waggoner
CEO, Co-Founder
Tayara Calina
Program and Development Manager
Anita Hernandez
Communications Coordinator
Ashlei Brittany
Conference Coordinator
Board of Directors
Christine Waggoner
President, Co-Founder
Dawn Blessing, MBA
Vice President
Kylie Harrison, MS, RDN, CLC
Secretary
David Law, MD
Board Member
BingYune Chen, MPH
Board Member
Vanessa Rangel Miller, CGC, MBA
Board Member
Rafael Escandon, DrPH, PhD, MPH, HEC-C
Board Member
Volunteers
Cara M. Weismann, PhD
GM1 Data Sharing Team Member
Rajiv Raman
Volunteer
Douglas Dooley
Co-Founder, Volunteer
Advisors
Kathleen Kirby
Scientific Advisor
Jeanine Jarnes, PharmD
Scientific Advisor
Samantha Parker, MBA
Scientific Advisor
Simon Jones,MBChB BSc MRCPCH
Scientific Advisor
Mark de Souza, PhD
Business & Operations Advisor
David Weinstein, MD, MMSc
Clinical Advisor
Philip Reilly, MD, JD
Scientific Advisor
Curtis Scribner, MD
Regulatory Advisor
Emil Kakkis, MD, PhD
Scientific Advisor
Paul Fitzpatrick, PhD
Scientific Advisor
Gouri Crommie, PhD
Scientific Advisor
Chris Phillips, MS
CMC Advisor
We’re Changing
GM1 Research Globally
Our work and advocacy contributes to brings increased awareness, scientific knowledge, and financial investment.
34+
PARTICIPANTS IN TRIALS
4
CLINICAL TRIALS TO DATE
9
SCIENTIFIC PUBLICATIONS
2
PUBLICLY AVAILABLE MOUSE MODELS