With research there is hope
With hope there is life
Welcome to the website of 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 at via email!
Stories and inspiration
Our community is full of vibrant people living life to the full! Learn more about our stories, and maybe take part in one yourself.
Information on ML
Information for people living with mucolipidosis, and for anyone else seeking to know more. Information for clinicians can also be found here.
About us
Cure Mucolipidosis is a 501 C 3 not-for-profit organization established in May 2021 with a single focus - to cure mucolipidosis!
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
$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
Are you here for 10,000 for 10? Thank you.
Will you be one of the 10,000 families that helps to cure ML?
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
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“I love dancing and singing. My favourite food is sushi and I love chocolate!”
Daisy, age 8
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“My favourite subject at school is art. I love being at home with my dog”
Jesse, age 12
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“My favourite place to go is the beach. I love watching movies and eating M&Ms”
Callum, age 11
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“Our boys are sweet and happy. We would love to find a cure for them, and for all the other kids”
Joshua and Declan, age 3 and 1
$10/Month
Help us change their life.
100% of contributions marked for research, go to research.
