Natanel Gold wants the gift of Gene Therapy for his Bar-Mitzvah
$92,011 raised
131% of $70k goal
581 contributors
162 Days running

Hello, I am Natanel Gold and I want you to meet the most special person in my life, my amazing 11 year old sister Shaindy. She loves music, pancakes, and of course ice cream. Shaindy has the most wonderful laugh, and just listening to her giggle makes me happy.

When she was 18 months old Shaindy was diagnosed with Mucolipidosis Type IV (ML4). Shaindy is unable to walk or talk, her brain works differently than ours, she is almost blind, and she will have a shortened life span.

Instead of giving me a gift for my Bar-Mitzvah it would mean much more to me if you would please make a generous donation to the ML4 Foundation to fund the ongoing ML4 gene therapy research.

Researchers at Harvard have already proven that gene therapy can work for ML4. These researchers are now working to bring gene therapy to Shaindy and other kids with this disease.

The ML4 Foundation’s mission is to fund, support, and promote medical research dedicated to developing treatments for Mucolipidosis Type IV (ML4), a genetic neurodegenerative storage disease.

Your donations directly support treatment-focused research at Massachusetts General Hospital, The Weizmann Institute, and other top laboratories around the world working on creating drugs to control neuro-inflammation and to create gene therapy for children with ML4.  You have a direct impact when you donate to ML4!

Your generous donations fund incredible research creating treatments for children with ML4. With dramatic cuts to NIH funding in recent years, researchers turn ever more to private foundations such as the ML4 Foundation for funding to keep their labs working on this disease. You can read more about the scientific research you are funding at We are particularly excited about a FDA-approved drug we are testing to control neuro-inflammation and restore function of neurons in the brain, and also about development of a gene therapy for ML4 that has already produced exciting data! Your donation helps this work succeed!

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