Our Mission & History of the Foundation

Our Mission

The ML4 Foundation is a non-profit corporation founded by families whose lives have been affected by ML4.

The mission of the Foundation is to fund, promote, and support medical research dedicated to developing effective treatments and ultimately, curing ML4 by

Providing critical financial support to researchers to pursue scientific endeavors related to ML4;
Linking individuals with ML4 with researchers for clinical studies;
Encouraging established researchers to stimulate interest in ML4 at high academic levels;
Sponsoring symposia and other programs concerning Ml4 research to inspire cooperation, communication, and collaboration among scientific researchers, academic institutions, charitable organizations, governmental agencies, and concerned individuals;
Raise awareness in the medical community to decrease the undiagnosed and misdiagnosed cases of ML4, and
Increase education in the Jewish community, and the general community, as to the availability of the carrier and prenatal screening tests.

History of the ML4 Foundation

The ML4 Foundation, originally known as CHARM (Children's Association for Research on Mucolipidosis), was founded in 1982 by Ron & Lynn Goldblatt, their parents, Roz and Joe Zakutinsky, other family members, and close friends.

In 1984 with the diagnosis of their daughter Lauren, Jordan Glaser and Randy Yudenfriend Glaser joined CHARM. Shortly afterwards the name of the organization was changed to the ML4 Foundation. Randy was elected President and served for over 20 years, until January 2009. Paul Tanenholz is now the current President. Jordan continues to serve as Medical Director.

ML4 was unheard of years ago. Most doctors did not know of this disorder. After exploring many avenues and researching whatever publications were available, we came across Dr. Gideon Bach - the head of the Human Genetics Department of Hadassah Hospital in Jerusalem, Israel. At that time, he informed us that there were 12 reported cases in the world. Since then, it has grown in numbers to almost a hundred reported cases, but doctors estimate there are many more times this number not diagnosed or misdiagnosed as cerebral palsy.

Medical research was the major reason we started the organization. In addition, we wanted to reach out to other families affected with this debilitating disorder.

P19 Gene Identified

ML4 gene discovered independently by Dr. Gideon Bach et all at Hadassah Medical Center in Israel and by the collaboration of Dr. Susan Slaugenhaupt's team at Harvard/Massachusetts General Hospital and Drs. Ehud Goldin and Sun Mei at the National Institutes of Health. The ML4 gene is a new protein that is probably an ion channel, but it's exact function is not yet known.

The finding of this gene will facilitate the identification of patients and will help perform efficient prenatal diagnosis and carrier testing. The identification of chromosome 19 led the way.

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.

Mucolipidosis IV Foundation

Our Mission & History of the Foundation