Play

Help Advance
Research and Advocacy for
GM1 Gangliosidosis

Children and families are in truly desperate need of help, facing a fatal degenerative disease with no treatment. The lack of approved treatments for GM1 is due to lack of funding. The knowledge and the science already exist to help. Together, we can advance and navigate the drug development process.
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.

DONATE NOW CONTACT US

$5M+

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

Rare Disease Day

Raising awareness and generating change for the 300 million people worldwide living with a rare disease, their families and caregivers.

  • May 23rd Annually

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.

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

Run, walk, bike, and hike for GM1 awareness and raise funds for our critically important cause.

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.

Take Action

Caregiver Support Group

Friends of Cure GM1 Group

About GM1​

Subscribe to Newsletter

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.

Read our Newsletters

Read about Advancements and Research

DONATE NOW
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

Christine Waggoner

Co-Founder, President

Christine Waggoner and her husband Douglas Dooley founded the Cure GM1 Foundation in April 2015 in honor of their daughter Iris and all those affected by GM1 Gangliosidosis. Christine received the Sanofi TORCH award in 2017 for outstanding patient advocacy and she also received the WORLD Symposium Patient Advocacy Leadership Award in 2023.  Founding and running Cure GM1 is a true labor of love to help bring a treatment to all those affected by GM1 Gangliosidosis. Read an in-depth interview is here.

Dawn Blessing, MBA

Vice President​

Dawn Blessing is Chief Business Officer at Neucore Bio and was recently Vice President of Scientific Strategy and Corporate Development at Zogenix. Prior to Zogenix, she served as Senior Vice President Corporate Development at Modis Therapeutics and previously Vice President, Corporate Development at Audentes Therapeutics. She has 30 years of experience in biotechnology finance, business development, and alliance management. Over this period, Ms. Blessing has focused on programs for rare diseases and the application of genetic information to drug development. Before joining Audentes, Ms. Blessing served as Senior Director, Business Development and Alliance Management at 23andMe, and Director of Business Development at BioMarin Pharmaceutical.

Kylie Harrison, MS, RDN, CLC

Secretary

Kylie lives in Colorado with her 4 daughters and husband, Kyle.  In June of 2020 her two oldest daughters, Kinley and Kennedy, were diagnosed with Juvenile GM1.  Since learning of her daughter’s diagnosis, she and Kyle have dedicated much of their time to raising awareness and funding to advance treatments for all children with GM1. Kylie recently became a Certified Nursing Assistant and is now paid to take care of Kinley and Kennedy.  The Harrison family has been fundraising to support research and development of treatment options for this horrific disease since the girls were diagnosed in 2020. To date they have raised over $350,000 for the foundation.

David Law, MD

Board Member

Dr. David Law is the father of Violet Aurora and Derek Sison Law. Violet was diagnosed with late-infantile GM1 Gangliosidosis and passed away at age 4. He is an internal medicine physician who trained at the UC Davis Medical Center and Harbor-UCLA Medical Center, and currently practices hospital medicine and palliative care medicine in southern California. He has been married to his wife, Dr. Veena Sison, a developmental pediatrician, since 2014 and together they enjoy traveling, attending live music events, and are the co-founders of 3rd Call Productions, an award-winning independent film studio in Los Angeles. They use the majority of their time now advocating for rare disease awareness and research.

BingYune Chen, MPH

Board Member

BingYune Chen is a healthcare thought partner and data scientist. He has decades of experience as a founder, startup investor, Fortune 10 manager, and nonprofit leader. By day, he leads data concierge services at BingYune & Co, an advanced analytics firm based in San Francisco, California. The firm builds data products at the intersection of strategy, technology, and inclusion to improve the performance of organizations and mitigate the bias in AI systems. BingYune earned his Master of Public Health and Bachelor of Science in Bioengineering from the University of California, Berkeley. He is passionate about partnering with leaders and teams to take collective, positive actions to ensure their organizations become a driving force for social, environmental, and economic benefit. In his free time, BingYune can be found sipping a kale smoothie or meditating on a rowing machine.

Vanessa Rangel Miller, CGC, MBA

Board Member

Vanessa Rangel Miller is certified genetic counselor, with over 15 years’ experience in rare disease, patient engagement and genetic programs. Vanessa is currently in Medical Affairs at Ultragenyx. Previously at Invitae, Vanessa served in Biopharma/Advocacy Business Development, bringing together multiple partners in the biopharmaceutical industry to launch genetic diagnostic programs. As a co-founder of PatientCrossroads (d.b.a. AltaVoice), Vanessa brought together technology, genetics, and external partners, and was responsible for >50 patient-centered registry programs in rare and non-rare disease. Vanessa has previously worked for Emory University, and completed her master’s in genetic counseling at the University of North Carolina-Greensboro and her MBA at Emory University.

Rafael Escandon, DrPH, PhD, MPH, HEC-C

Board Member

Rafael “Rafa” Escandon  has worked in global clinical research and development and patient advocacy in the biotechnology industry for three decades and is currently a consultant to the biotechnology industry.  His approach is to be highly considerate of the ethical implications of conducting and participating in clinical research, especially with advanced therapies in rare conditions, as well as in vulnerable and developing-world populations. He has been a member of the NYU Grossman School of Medicine’s Department of Bioethics Pediatric Gene Therapy Medical Ethics Working Group (PGTME) since 2019, and is a contributing graduate faculty member in Pediatric Bioethics at the Children’s Mercy Bioethics Center in Kansas City Missouri. Rafa has also served as adjunct faculty at the University of California Berkeley’s Clinical Research Management Program and as an educator in human rights and justice. He holds graduate degrees from the University of Maryland at Baltimore, Walden University of Minnesota, and the Johns Hopkins Bloomberg School of Public Health, and is a certified healthcare ethics consultant.

Cara M. Weismann, PhD

GM1 Data Sharing Team Member

Dr. Weismann is the Director for the Program of Excellence (POE) in Mucopolysaccharidosis (MPS) and Lysosomal Storage Disease Advanced Therapies at the non-profit organization the Orphan Disease Center. Within the POE, she leads a grant program whose goal is to accelerate the development of therapeutics and provide scientific resources to the MPS and lysosomal storage disease communities. She has extensive experience in therapeutic research, drug development, business development, and data sharing policy. She is passionate about using these skills to bring treatments to rare disease patients throughout the world.

Anita Hernandez

Program and Outreach Coordinator

Anita joins the Cure GM1 Foundation as the Program and Outreach Coordinator, infusing her expertise in creating captivating digital experiences with a passion for making a tangible impact. With a robust background in client relations, brand identity, and digital strategy honed at Love Hope Design, Anita has excelled in leveraging digital marketing to support non-profits and mental health initiatives. Her strategic vision and holistic approach are pivotal in our mission. At Cure GM1, she aims to harness this power to enhance awareness, engage the community, and drive support towards finding a cure for GM1 gangliosidosis

Rajiv Raman

Volunteer

Rajiv Raman is a seasoned full-stack engineer and digital artist with 20+ years of experience in software development and UI/UX design. Proven ability to blend technology and creativity to deliver captivating digital experiences. A visionary problem-solver, passionate about pushing boundaries and creating impactful art in the digital realm and physical world.

Douglas Dooley

Co-Founder, Volunteer

An animator by training and an innovator in the field of human robotics interaction, Doug uses his free time and professional skills in animation, computer graphics, and graphic design to support the foundation’s work in honor of his daughter Iris who suffers from juvenile GM1 gangliosidosis.

Advisors

Kathleen Kirby

Scientific Advisor

Kathleen Kirby has over 30 years of drug development experience in Clinical Development Operations and Program Management.  She is well versed in strategic planning and preparation of product / clinical development plans, and establishes a bridge between program development, clinical strategy and clinical trial execution in early phase start up companies focusing on gene therapies, rare diseases, patient recruitment strategies and natural history studies.  Kathleen made significant contributions during early phase development of SKYSONA (eli-cel) in Cerebral Adrenoleukodystrophy and ZYNTEGLO (beta-cel) in Beta Thalassemia. Kathleen supports Cure GM1 and other rare disease foundations.

Jeanine Jarnes, PharmD

Scientific Advisor

Jeanine Jarnes, PharmD, is an Assistant Professor in the University of Minnesota Department of Pediatrics and is an adjunct assistant professor in the College of Pharmacy, University of Minnesota. Jeanine is a board certified Pharmacotherapy Specialist, as well as having a board certification in oncology pharmacy. Jeanine works as a pharmacotherapy provider for patients with inherited metabolic diseases at the University of Minnesota Specialty Clinics. She also serves as a Clinical Pharamcogeneticist for the Lysosomal Disease Network and as a clinical researcher for patients with lysosomal diseases.

Samantha Parker, MBA

Scientific Advisor

Samantha Parker has over two decades of rare disease healthcare experience in the biopharmaceutical industry. She has a proven track record in patient advocacy, gene therapy development, natural history studies, registries, novel patient-centered outcomes, early access planning and collaborative networks. Samantha was a founding member of several pilot EC European Reference Networks – virtual networks of healthcare providers aiming to facilitate discussion on complex or rare diseases. Samantha is an adamant believer that patients should be at the front and center of rare disease research and development. She is currently vice-chair of the international rare disease research consortium (IRDiRC), aimed at promoting international collaboration and advance rare diseases research worldwide.

Simon Jones,MBChB BSc MRCPCH

Scientific Advisor

Dr Simon Jones is a consultant in pediatric inherited metabolic diseases at the Willink Unit at Saint Mary’s Hospital.

His major research interest is therapy for lysosomal storage diseases (LSD’s).

He received his medical training at the Edinburgh University Medical School, Edinburgh, UK, with a BSc in Neurosciences. He moved to London and trained in Paediatrics at Guy’s and St. Thomas’ Hospital, London, UK.  He has been working at the Willink Biochemical Genetics Unit in Manchester since September 2005. Since 2008, he has been a consultant in pediatric inherited metabolic diseases at the Willink Unit and is now the clinical lead for the LSD service. The Willink Unit is now part of the Manchester Centre for Genomic Medicine at Saint Mary’s Hospital.

Mark de Souza, PhD

Business & Operations Advisor

Mark has over 20 years of executive and entrepreneurial experience in the rare disease space at multiple venture backed companies. He held positions of increasing responsibility in business development (culminating as VP) at Dyax Corp, which was acquired by Shire for its FDA-approved drugs for hereditary angioedema. He has been a founder, CEO, or Executive Chair of Lotus Tissue Repair (a Third Rock Ventures company acquired by Shire), PellePharm and Molecular Skin Therapeutics (both BridgeBio companies), SeylanMed and Chromaderm (both merged into Dermbiont), NFlection Therapeutics, and Phoenicis Therapeutics. Mark is a board member of SNAPkids, a San Francisco Bay Area non-profit organization, and an advisor to the CureGM1 Foundation.

David Weinstein, MD, MMSc

Clinical Advisor

Following his graduation from Harvard Medical School, Dr. Weinstein did a residency, chief residency, and
fellowship in pediatric endocrinology at Boston Children’s Hospital.  He subsequently obtained a Masters
in Clinical Investigation from Harvard and MIT, and became Director of the Glycogen Storage Disease
Program at Boston Children’s. In 2005, Dr. Weinstein moved to the University of Florida where he
directed the GSD Program and became a tenured professor.  He moved to the University of Connecticut
in 2017 where his team launched the world’s first gene therapy trial for glycogen storage disease. In
2020, he left the academic world to serve as the medical lead for the GM1 gangliosidosis and
metachromatic leukodystrophy gene therapy trials at Passage Bio. He subsequently served as acting
Chief Medical Officer for Grace Science, LLC leading the submission of the IND for gene therapy for
NGLY1 deficiency, and he remains a consultant for rare disease trials.
In 1989, he was named as one of the inaugural Goldwater Scholars.  He is a former Jan Albrecht Award
winner from the American Association for the Study of Liver Diseases, and he was the George Sacher
Award winner from the Gerontological Society of America.   In 2013, Dr. Weinstein was knighted in
Poland and honored with the Order of the Smile international humanitarian award.

Philip Reilly, MD, JD

Scientific Advisor

Phil focuses on creating and building companies that develop breakthrough therapies for orphan genetic diseases. He is passionate about working with patient groups, and helping to unite those suffering from rare diseases in order to foster a support network and potentially improve patient outcomes.

Phil has authored or co-authored more than 100 articles in scholarly journals and has published seven books including most recently Orphan: The Quest to Save Children with Rare Genetic Disorders. Phil currently serves on the Advisory Board to the Boston University School of Public Health, and has served as a trustee emeritus of Cornell University, an Overseer of Weill Cornell Medical College.

Emil Kakkis, MD, PhD

Scientific Advisor

A medical geneticist by training, Dr. Kakkis is currently founder and CEO at Ultragenyx, a
biopharmaceutical company developing novel products for the treatment of rare and
ultra-rare diseases. The company was founded in 2010 and went public in 2014, and has developed three approved therapies (Crysvita, Mepsevii, and Dojolvi) and now holds ex-US rights to a fourth approved product (Evkeeza), and a deep pipeline of potential treatments across a variety of therapeutic modalities including gene therapy, mRNA
therapy, biologics, and small molecule.

Gouri Crommie, PhD

Scientific Advisor

Gouri Yogalingam-Crommie has almost 30 years of academic and industry experience in the lysosomal storage disease field, and is currently working as a Director of External Collaboration in the Cardiovascular Research group at BioMarin Pharmaceutical Inc. Gouri has extensive experience with working on scientific strategy, and successful collaboration with cross-functional teams to develop safe and effective treatments for patients affected with lysosomal storage disease. Gouri has focused on understanding how inherited mutations lead to lysosomal storage disease in both patient cells and animal models of disease. She has used these cell and animal models of disease to investigate several therapeutic strategies, including gene therapy, enzyme replacement therapy, chaperone therapy and stem cell strategies for MPS VI, MPS IIIA, MPS IIIB, CLN2, GM1 gangliosidosis, and sialidosis.

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