Results of fundraiser for Milestones for Maxwell

The 2nd Annual Milestones for Maxwell Golf Tournament was held July 29 at Fossil Trace Golf Club. Amber Freed, Founder, CEO  &  mother was happy to report that the golf tournament raised $250,000. Amber said that $2,000,000 of the $4,000,000 required to advance a Phase One clinical trial was all raised via the GoFundMe and in thanks to the golf tournament.  

During the shutdown gene therapy stalled because scientists could not go into the laboratories to work. Every known FDA drug was tested against Maxwell’s mutation in a petri dish hoping  to lead to a new discovery.  In Dec. news was received that a drug named Ravicti, primarily used for urea cycle disorders, had a large positive impact on Maxwell’s disease so a trial was organized. Maxwell was the first patient in August at Weill Cornell in New York City. Ravicti costs $850,000 a year but the manufacturer has donated a year’s supply. Maxwell is showing signs of improvement; he has less body stiffening, less eye rolling and his developmental trajectory has improved.

Maxwell and twin sister Riley love Pez dispensers but they always broke my heart because Maxwell did not have the fine motor skills to retrieve the candies, according to Amber. Amber and her husband Mark both cried. Amber said that they feel like they are finally getting to know their beautiful son and Children’s of Colorado has championed their cause and they were so proud to write a giant check in order to sponsor the trial. SLC6A1 has been liked with Parkinson and they are hopeful the gene therapy that is being created will benefit the Parkinson’s community also. Their goal is to begin the gene therapy trial within the next 18 months. 

Amber said, “the drug is not a cure – it is not gene therapy – but it has the potential to buy Maxwell time”. Amber said that there are no guarantees in life, but that they would not made it this far without media coverage, key introduction, donations, Facebook birthday fundraisers, silent auction, golf tournaments, etc.  

Amber said, “the gene therapy treatment is progressing spectacular. It’s a when – not an if. They are moving at the speed of light years for science and I’m very hopeful it will be in time for my little man, Maxwell.”