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


Gene Therapy Advances

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The ML4 Foundation is funding several projects in gene therapy. One of these projects involves a collaboration between Dr. Steven Walkley (Einstein) and Dr. Charles Venditti (NIH). Read here about…


CoRDS patient registry a success!

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  Terrific enrollment by ML4 families in our CoRDS patient registry! In 2015, the ML4 Foundation launched our disease-specific patient registry with Sanford CoRDS. This registry contains clinical records by…


Newly Funded Research

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The ML4 Foundation Board of Directors is pleased to report approval of funding for Dr. Christian Grimm (University of Munich) for his proposal, Known and novel members of the endolysosomal…

Sarah Zar Purim

Exciting New Investigations at biotech startup, Perlara PBC!

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The ML4 Foundation is pleased to announce that Perlara PBC, a Bay Area biotech created to discover personalized cures for diseases rare and common will begin research on Mucolipidosis Type IV. Perlara (…