Make a gift in honor of Sophie today!
(Note that donations made through this form are in U.S. dollars)
Sophie is now 24 years old. She was born with ML4. Somehow, both her dad and I gave “affected” copies of our Chromosome 19 alleles to Sophie, “affected” which we didn’t even know lurked within our DNA. Now, Sophie gets to live with the result.
There are three stages of ML4. The first is where the children gain skills, very slowly, but still there is a gain. This occurs in the first decade of life. The second stage is where the children slowly lose eyesight, but do not gain any new skills – they hold steady. The third stage occurs in the late teen, early adult years where skills get lost, muscle stiffness increases, so much so that many times hands curl up and tight muscles pull the spine into scoliosis. Muscle pains increase – have you ever had a “Charley Horse”?
We are increasingly seeing the third stage of this devastating disease begin to appear in Sophie. Through previous fundraising efforts, a treatment has been developed to halt the progression of the symptoms, but additional funding is necessary to get the treatment to the manufacturing stage.
Please consider helping to fund these necessary treatments for Sophie and others affected by ML4.
The Bicknell Family
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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 Sophie Bicknell.
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
