The CURE GM1 FOUNDATION’s mission is to fund research for the benefit of all those who suffer from GM1 gangliosidosis. The foundation has extremely minimal overhead costs with the maximum amount possible being allocated towards advancing treatments. This nonprofit organization was founded by parents of children who suffer from GM1 who seek to save the lives of all those who suffer from this wretched condition. The Cure GM1 Foundation is dedicated to directly funding research for a cure for GM1 gangliosidosis – a lysosomal storage disease that attacks the brain and spinal cord, and is always fatal in children. GM1 is a progressive and degenerative condition with an extremely broad and debilitating array of symptoms and complications.
Cure GM1 Foundation的外部链接
US，CA，Albany，94706
We are grateful to GEMMA Biotherapeutics and James M. Wilson, MD, PhD for continuing to work on GM1 gangliosidosis.
James M. Wilson, MD, PhD founded two biotechs, GEMMA Biotherapeutics and Franklin Biolabs, to prove that gene therapies could be good business. “I think what we’re seeing now is more than a correction, and I’ll coin the term ‘irrational pessimism,’” Wilson said at #STATBreakthrough.
We are tremendously grateful to Cat Lutz and her team for helping to create the first publicly available mouse models for GM1 gangliosidosis. READ MORE: curegm1.org/mousemodels #curegm1 #raredisease #hope #science #research #charity #genetics
Flowers, bulbs, gardening supplies and more to help fund research, drug development, and awareness! SHOP NOW: curegm1.fpfundraising.com #curegm1 #raredisease #fightforlife #hope #community #donate
The Cure GM1 Foundation supported the preclinical research to assist in moving an intravenous AAV9 gene therapy forward to the clinic. Newly diagnosed children are still enrolling in the trial at the National Human Genome Research Institute (NHGRI) today. ABOUT: curegm1.org/about-gm1 #curegm1 #research #genetherapy #raredisease #medicine #genetics #aav #donate #takeaction
Now is the time to reach out to your governor, mayor or government officials to recognize GM1 Day in May. Awareness->Funding->Hope. ABOUT: curegm1.org/gm1day CONTACT: [email protected] to take part and to help #curegm1 #hope #raredisease #awareness #fundraising #donate #charity
Our work must continue so that children and all those ineligible for trials will have a chance at life. Will you join our fight? We all have the capacity to help. TAKE ACTION: curegm1.org/take-action #curegm1 #raredisease #nonprofit #medicine #biotech #genetics #invest #donate
We are thankful to GEMMA Biotherapeutics and to Rojan Vakili for her advocacy on behalf of Darian and all those suffering from GM1 gangliosidosis.
I was so honored to be asked by GEMMA Biotherapeutics to share Darian’s story on Rare Disease Day. I spoke about Darian’s diagnosis, clinical trial experience and what he has had to over come in his short life. My hope is that I highlighted the human aspect of their research to their 200+ employees. I will never tire of talking about my beautiful boy.
Did you know that Cure GM1 was approved to be the only 501(c)(3) entirely dedicated to GM1 gangliosidosis advocacy, research, drug development and awareness 10 years ago! Our anniversary is coming in April and this fight for life takes tremendous tenacity. We are proud of the strides and progress. Join us this year as we celebrate our 10th anniversary year! LEARN MORE: curegm1.org/about-gm1 #curegm1 #raredisease #patients #community #genetics #research #advocacy #give
The Cure GM1 Foundation’s Annual GM1 Community Conference is an invaluable resource for our community, researchers, and anyone interested in rare diseases! This is a hybrid event with virtual and in-person options. Registration is now open and is FREE for families! JOIN US: curegm1.org/conference If you are interested in sponsoring the event or have questions, please mail [email protected] #curegm1 #raredisease #nonprofit #advocacy #community #genetics #charity #donate
"No parent should hear that their child has a disease that has no treatment or cure." -Milah's mom STORY: curegm1.org/milahs-story We are absolutely heartbroken for sweet Milah's family at this time of tremendous loss. #curegm1 #childloss #grief #raredisease #nevergiveup #charity