The Nicoara Family

Make a gift to The ML4 Foundation in honor of Rose today!

Hello from the Nicoara family!

As most of you know, our daughter Rose was diagnosed a few years ago with Mucolipidosis Type IV (ML4), an extremely rare disease that affects all areas of her development, including her ability to walk, talk and feed herself. Even though she has these many seemingly insurmountable challenges, Rose is one of the happiest kids we know. She is always smiling, and loves being around other people. If there is music, dancing and lots of people, Rose is as happy as can be. Though we are so thankful that she is happy and otherwise healthy, we also know that ML4 is a disease that will get progressively worse over time, and Rose will slowly lose the skills that she has now. The ML4 Foundation is closer than ever to having a gene therapy approved for the treatment of ML4. Please help in any way you can, and share this campaign with others. We are so grateful for all of your support.

Thank you so much!


ML4 is a genetic disease caused by the mutation of a single gene that results in the loss of a protein (MCLN1). This incredibly important protein is required by the body for normal functioning: without MCLN1, children and adults with ML4 suffer from progressive and debilitating loss of gross and small motor function, go blind, develop problems chewing and swallowing, rarely have the ability to speak, cannot walk, and over time, lose many abilities that they work hard to acquire when they are young.

The funds raised by the ML4 Foundation are directed to a research program at Massachusetts General Hospital in Boston. We have been working for several years with a team there to create a gene therapy to treat ML4. We are also funding a large-scale natural history study of ML4, bringing ML4 patients from around the world to the MGH clinic to be seen by specialists in this disease. YOUR DONATIONS made this work possible and allow it to continue at a speedy pace!

If you prefer to donate by check, you may mail it to the following address, and note that it is given in honor of Rose Nicoara.

Mucolipidosis IV Foundation
1440 Spring Street NW
Atlanta, Georgia 30309

The ML4 Foundation is a U.S. 501 (c)(3) nonprofit charitable organization.
Tax ID 13-3633501