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Mucolipidosis Type IV:
A Devastating Genetic Disease

Mucolipidosis Type IV (ML4), was first identified in 1974 as a genetic disease. Children with ML4 begin to exhibit developmental delay during the first year of life. Many parents seek ophthalmologic evaluations for pseudo-strabismus and medical intervention for delayed motor milestones. Children with ML4 typically reach a maximum developmental age of 18 months in language and motor function, although their receptive abilities are more advanced.Read more


Collaboration Toward a Cure 
In June 2013, 20 international researchers met for 2 days in one room to share, debate, and discuss the current state of information about MLIV science. The meeting led to new collaborations, new understandings of the disease, and was electrifying for all present. View this video from the conference.
 
Why Fund Rare Disease Research? 
Since ML4 disease is so rare why should we urge for funding in this disease?  Listen to how the scientists explain why funding in rare diseases like ML4 is critical, not only for solving the devastating needs of children with this disease, but for making discoveries in common diseases, as well. Their insights will change how you think about funding for ML4.

Do For ML4We all "Do" every single day. Whether we read a book, go to the gym, play golf, clean the garage or learn a new skill, we are always "Doing." What better way to raise money to help find a cure for ML4 than to "Do for ML4"?  Join us in raising money for medical research and create your own fundraiser centered around the things you already "Do!" It’s easy and fun to ask your friends to support you while you "Do For ML4"!


  

 

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Do for ML4
Jewish American Hero
Randy Gold, ML4 Foundation Vice President, was named the 2011 Jewish Community Hero sponsored by the Jewish Federations® of North America for his efforts around promoting awareness of the 19 known preventable Jewish Genetic Diseases.
Jewish Genetic Disease Consortium National Organization for Rare Disorders JScreen