The mission of the Foundation is to fund, promote, and support medical research dedicated to developing treatments and a cure for ML4.


Your donations directly support ML4 researchers at Harvard, Einstein, Baylor and other top academic institutions dedicated to finding treatments for Mucolipidosis Type IV.


What is ML4?

Mucolipidosis Type IV was first identified as a genetic lysosomal storage disease in 1974. Children with ML4 begin to exhibit developmental delays during the first year of life and many parents often pursue ophthalmologic evaluations for pseudo-strabismus.

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Our Mission

ML4 is a genetic disease causing mental retardation, retinal degeneration, gross and small motor dysfunction, and limited lifespan. The Foundation’s mission is to fund, support, and promote medical research dedicated to developing treatments and a cure for ML4.

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Patient Registry

If you or a family member have been diagnosed with ML4 or have not yet received a diagnosis, you are invited to participate in the CoRDS registry. Connect to the exciting science news and receive notifications of research opportunities for which you are eligible!

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The Latest News

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Welcome to the new ML4 Foundation website!

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We are super excited to launch our new ML4 Foundation website! We have been working on this site for a few months and this clean, modern new look has been…


Spearheading committee with NORD on older patients

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I have heard from some parents of ML4 young adults that they would benefit from resources to help families with children as they transition from late childhood and into adulthood with…


Add to our Gallery

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  Please be sure to visit our Gallery of Images to see many gorgeous pictures of our children. I am always eager to add to this collection of beautiful pictures. If you…


University of Pennsylvania request for proposals in MLIV

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So many of our ML4 families and supporters worked hard to raise $25,000 funds for the UPenn Center for Orphan Disease Research and Therapy so the ML4 Foundation would secure…