$4M Needed for Lifesaving GM1 Enzyme Replacement (ERT)

I first became aware of the terminal disease, GM1 gangliosidosis through a personal relationship with a sweet girl named Iris. I first met Iris while I was volunteering at SNAPkids (Special Needs Aquatic Program). My father who has worked in biotech for decades also volunteers at SNAP. We started swimming with her through SNAP, her smile lit up the pool and she was always cheerful and fun to interact with. However, I have seen the debilitating effects of this disease firsthand on my friend and she, other children, and babies are dying due to GM1.

GM1 gangliosidosis (GM1) is a rare inherited genetic disorder that primarily affects babies and children. It has many debilitating and truly devastating ramifications, some of which are developmental regression, mobility deterioration, seizures, visual impairment, and neurodegeneration. This is a fatal disease that currently has no cure or effective treatment. Thousands of people like Iris suffer from GM1 and face the progressive loss of function and abilities until death.

Fortunately there is hope and something that can be done. The Cure GM1 Foundation was founded to research and to help develop possible treatments for this disease. The Cure GM1 Foundation has supported many projects and initiatives over a ten year history, but enzyme replacement therapy (ERT) has never made it to clinical trials for GM1. ERT is a very well-known and reliable mode of treatment. Cure GM1 has already raised millions to support research and drug development. The funds raised will be going towards IND-enabling studies, tox studies, and tox material manufacturing. The Cure GM1 Foundation has already selected contract development manufacturing entities and is working to gather proposals for the studies to be completed by experts but funds are essential to further developments.

However, an additional $4 million is necessary to complete the enzyme manufacturing process. Time is of the essence! Any and every contribution furthers the treatment of some of these kids extending their lives. If you cannot donate, then please consider sharing this. Imagine if your child or loved one in your family had a similar situation. Families are told their child will die because of a lack of funding. Surely their lives are worth fighting for. By raising awareness and funds to support the foundation, we can help further enzyme replacement and attract investment and ultimately, help dying babies and children.

I’m so excited to work with the Cure GM1 team. They have advisors, physicians, and scientists who have worked decades on lysosomal storage diseases like GM1. They have worked very hard to raise seed funding, but more funding is required. You have the power to help save these kids lives. Please help me make a difference.

More details about GM1 gangliosidosis on the CureGM1 Foundation’s website: curegm1.org/about-gm1