July 6, 2017

We're thrilled to announce a new partnership that will bring our resources in front of The Mighty's wide-reaching readership. We will now have a growing home page on The Mighty and appear on many stories on the site.

June 6, 2017

We were so pleased to work with the Washington DC based filmmaker Rachel Feichtner of Brave Girl Productions on this video titled "Reframing Hope."  So many people have questions about what it means to care for a child with a life-limiting condition. We wanted to tell...

October 23, 2016

Not long ago, I found myself speaking to a program administrator at the FDA. It was a classic elevator pitch, I told him all about the breakthroughs in gene therapy for leukodystrophy, specifically, MLD. My voice cracked with emotion recounting how I had recently told...