Racing to a Cure

May 9, 2019 at 1:04 am Leave a comment

I got an email recently from Mark Freed ’97, an acquaintance from my undergraduate days, that felt important to share here:

“My wife and I have twin children named Maxwell and Riley. In 2018, my son was diagnosed with a rare neurological disease known as SLC6A1. The disease is too rare for a formal name and is known only by its genetic location in the brain. Doctors told us we were 1 of 30 in the world and nothing could be done and we chose not to accept that answer. My wife and I interviewed 140 scientists over the course of three months and found the disease can be cured via gene replacement therapy. We are in the midst of raising a large sum of money to advance the clinical trial. We hosted the first symposium on the disease in November and found that the disease is dramatically under-diagnosed because it’s newly discovered. Our patient population is actually in the thousands . . . The quest to save our son has transcended our family and we have the opportunity to impact a multitude.”

Mark and his wife, Amber, are leading an effort to save Maxwell and other children affected by SLC6A1, with a goal of raising $1,000,000 to advance a gene replacement therapy clinical trial. It’s a daunting task, but the impact literally could be life-saving. They’re concentrating their efforts on a GoFundMe, and they’ve also launched a website with details on the disease, stories of Maxwell and other children afflicted, and updates on the research.

Ryan Jones, editor

 

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