Make a gift in honor of Tommy today!
This is Tommy, our son. He is twelve years old, and a few years ago, he was diagnosed with a rare genetic disorder: Mucolipidosis type IV (or ML4 for short).
ML4 is a degenerative lysosomal storage disease, which affects every single aspect of a child’s development, psychological and motor. It is extremely rare (only a few hundred cases worldwide, while Tommy is only one of five known in France, where we live) and our understanding of its effects, both short and long term, are limited.
The major impacts are neurological and visual. Most of the children who have the disease never walk, never talk, lose their sight in the teen years and have a lowered life expectancy. After a double corneal graft when he was younger, Tommy recently had to have a cataract operation.
Most of the confirmed cases are in the United States, so a foundation was set up in Atlanta by families of the children, devoted to understanding ML4 and funding research into treatment. See section below for more information about The ML4 Foundation.
Over the last 4 years, the money we have raised has financed a retrospective natural history, helped to define biomarkers and to make huge advances towards gene therapy. The results already achieved have been sufficient to give us a real shot at clinical trials and a possible therapy. But before that, there are more steps that need to be taken, including a major prospective natural history. All of this costs money. If you would like to help fund research for Tommy and other children like him, you can donate here. Any amount is helpful.
Thank you all so much for taking the time to read this message! We really hope you will participate even a tiny amount to help Tommy and others!
ABOUT THE ML4 FOUNDATION
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 Tommy Brown.
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