Every Donation Counts

In October 2020, Grace became the first MSD patient in the world to undergo a bone marrow transplant in hopes of finding a cure.

There have been fewer than 100 reported cases of MSD in the world. At the time of their diagnosis, our children were patients number 18 and 19 living with MSD in the United States.

MSD is a rare and terminal disease that makes the body unable to get rid of cellular waste, which eventually will contribute to a progressive loss of mental abilities and movement. Without a treatment, our children will become severely disabled, suffering dementia, losing their ability to walk, talk, eat, see, and hear. This will force us to make a decision that no parent should have to make. Outwardly our children look healthy, but their body is broken on the inside. Typically, children with MSD only survive a few years after the signs and symptoms of the condition appear. Sadly, MSD children often die before their tenth birthday.

Rare diseases impact more lives than cancer and AIDS combined, yet 95% of rare diseases lack a FDA approved treatment. There are a small number of research studies going on throughout the world for MSD at this time. Currently, scientists can cure MSD in mice. Funding has been the #1 barrier in pushing treatment options beyond research and into clinical application. Private donations have primarily funded research projects to date. We need funding to get MSD treatment into children. The progress made in developing treatment options not only touches our rare disease population, but is proving to have far reaching implications for other more well know conditions like Alzheimer’s and Parkinson’s disease.

Please help give our children a chance at life and help save other precious lives touched by this terrible disease.