Ataluren Results

Yesterday was really difficult.

In 1999 our Scientific Director Lee Sweeney published an article on Gentamycin and while that was good news, we knew Gentamycin could not be a lifelong therapy. Then PTC marched into our lives, committing time, talent, and millions of dollars. They paved a road where none had been before. They became friends, invested in our community and our sons.

Over the years, we watched as the words premature stop and PTC124 nearly became household words. We found ourselves hanging our heart on the concept of the first treatment for Duchenne, if only for a subset, knowing that we would do whatever it takes to expand our horizons until every boy had a treatment. PTC developed and executed the first pivotal study of a New Chemical Entity (NCE) in Duchenne.

This is a huge step in and of itself, recruitment completed two months ahead of schedule. Parents/families, physicians, industry all coming together from around the world. Duchenne was studied in a systematic way and more thoroughly than ever before.

The outcome we have received is devastating and the news yesterday threw all of us over the edge. This blow knocked the breath out of all of us and at least for a moment, some of the wind out of our sails. PTC will further analyze the data and we will learn more about the results and the trial.

I’m still hopeful we will see some light and with that, a path forward. Regardless, we are still believers in our sons, in our community, and in PTC.. There will be treatments for Duchenne and we will find them. That is certain. PTC has been building the road and will continue. They are not changing their commitment to our sons. They are acting in our best interests and will continue to stay focused on the road forward.

I just wish the road was a bit easier and without so many damn boulders.