With research there is hope.
With hope there is life.
Welcome to Cure Mucolipidosis!
Mucolipidosis is considered part of the Ultra-orphans of the Lysosomal Storage Disease family of which there are over 60 known different disorders.
We invite you to explore our website and learn more about what we are doing. Please join us on Facebook, follow us on Instagram or contact us via email!
Life changing research.
Gene therapy has cured diseases just like mucolipidosis. To save these children, we need to sponsor the scientists who create our cure.
Prototype drug creation:
Prototype drug creation: $50,000 goal achieved!
Prototype drug testing (safety trials):
$85,000 of $300,000 goal so far!
Regulatory review and approvals:
$500,000 goal
First trials in patients (Clinical trials):
$500,000 goal
What is Mucolipidosis?
Within the cell, there's a constant cycle of building and breaking down molecules. For this process to stay efficient, cells must regularly remove the byproducts or "waste" generated. In Mucolipidosis, there's a problem with this waste removal system.
How can I help?
For the first time, a cure is possible. Help us to help families just like yours.
If we can raise $2 million, we can save these children. Can you make a donation?
Make the cure possible
Help us change their lives for just $10/month.
100% of contributions marked for research go to research.