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Since Rose was a baby, we had suspected that something was not quite right with her development. She was late to sit, roll over and crawl. She wasn't talking or using her fine motor skills properly. She couldn't do all of the other normal things that babies and toddlers her age could do, and we didn't know why. We began seeing different doctors and therapists, and finally got some answers. In January of this year, after many tests and doctor's visits, we found out that Rose has an extremely rare genetic disease called Mucolipidosis Type 4. All aspects of her development and vision are affected by this disease, and as of right now there is no cure. But through the amazing ML4 Foundation, there is wonderful work being done in the best hospitals in the world to find treatments for different parts of the disease, and drug trials may be under way as soon as 2019. Your donation can make a huge difference in supporting further research. To design a robust treatment, scientists need excellent clinical studies of patients to understand exactly how the disease progresses. Not only do these studies provide valuable information to the researchers about possible treatments, but they are also essential for working with the FDA to enter clinical trials. Earlier this year, Rose and I traveled to Boston to take part in this study, and seeing firsthand what is being done was amazing and gave us great hope for her future.
Our family would greatly appreciate any donation, big or small, that you can make to this campaign. Thank you all for your love and support of Rose and of our whole family.
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 www.ml4.org. 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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