NC Science Award RJ Samulski Chris Jess Curran

April 7, 2021

N.C. Awards Committee
North Carolina Department of Natural and Cultural Resources
109 East Jones Street, Suite 315
Raleigh, North Carolina 27601

Re: Nomination of Dr. R. Jude Samulski for the 2021 North Carolina Science Award

Dear Awards Committee:

It is with great pleasure that Jessica and I write this letter of support for Jude Samulski, PhD. The amazing impact that he has had on our son, Conner, cannot be overstated.

Conner was born in September of 2010 and, having difficulty nursing, we were concerned from the start. He continued to have developmental delays throughout his early childhood, which we addressed with physical therapy. Despite our best efforts with the therapy, we became even more concerned that he was falling even farther behind. Conner was exhibiting frequent falls, difficulty with stairs, exhaustion from minor physical activity and, worst of all, he was not able to keep up with his classmates, often isolated and playing by himself. We made an appointment with a Developmental Pediatrician.

The diagnosis of Duchenne Muscular Dystrophy brought us to our knees. It was heartbreaking for us and devastating for Conner. We were told he would experience a painful and grueling continuation of his physical decline with walking to end first, then even the simple act of lifting his arms to hug us would end. Breathing without assistance would become impossible and, most devastating of all, he would not be expected to live past his twenties. He would experience an unforgiving, harsh decline ending in heart and lung failure. A fatal and terrible diagnosis. We were stunned and helpless in the face of this horrific disease. It is a parent’s worst nightmare to have to watch your child suffer and not to be able to do a single thing to help them. There was no hope in sight.

Unbeknownst to us at the time, there was hope in the form of R. Jude Samulski, PhD. We had no idea that this dedicated researcher would have such a profound impact on Conner’s life. In March of 2018, Conner was fortunate to be chosen as the first patient to be dosed with Pfizer’s mini-dystrophin gene therapy drug developed by R. Jude Samulski, PhD. We prayed that Conner would not experience any adverse reactions and we were relieved to find out that Jude would be at Conner’s side for the infusion. It was an honor for us to have him there and an extraordinary comfort to have him explain what was going to happen. We happily report that Conner is doing well at 3 years post infusion. His stamina and endurance improved considerably. He is able to walk farther than pre-infusion. His North Star Assessment tests improved dramatically during the first 2 years post-infusion - ranging from a 50% improvement to a 100% improvement depending on the test. Most Duchenne patients only experience decline, and we are incredibly grateful to Jude for providing Conner these years of muscle stability. It has been a blessing for Conner to be able to maintain so much function for such a long period of time. It has not only helped physically but emotionally as well. These past 3 years Conner has been able to interact with our family and his peers without extreme accommodations and we have cherished each memory that he was able to make. Recently, Conner has experienced some leveling off of his abilities, but we remain hopeful because Jude is hard at work developing methods that will enable Conner to be redosed with gene therapy. Currently, there is no method to redose without a catastrophic immune response to the AAV but we have faith that Jude will be able to overcome these obstacles.

We are so grateful to this brilliant man who developed an amazing gene therapy. Conner was extremely lucky to have had these last years of mobility and we are hopeful that Jude has quick success with his efforts in developing a redosing method so that Conner can continue to be mobile and to make some more awesome memories!

Sincerely,
/Christopher and Jessica Curran/