Tennessee Roundtable Recap

On Sunday, April 2, 2017 Jett Foundation hosted the Tennessee Educational Roundtable in Nashville. 6 companies developing drugs for the treatment of Duchenne attended and presented to families in Nashville.

  • Mary Frances Harmon from PTC Therapeutics presented on autaluren, a drug approved in Europe for treatment of Duchenne patients with a nonsense mutation and that is currently being review by the FDA.

We learned at the Nashville Roundtable, that for families living with Duchenne, even those with one or more clinical trial options available for their child, no decision is the right decision. Living with the diagnosis of Duchenne means living in a world of uncertainty, and that uncertainty is amplified when navigating difficult decisions about enrolling in investigational drug trials where there are burdensome medical procedures, travel, cost, placebos, and long term commitments are involved. 

“I want to put my child in a trial, but I am overwhelmed by the options and I don’t know enough about them and I don’t want to do the wrong thing.” 

“Wow, in that one you need biopsies, but in this one we would have to travel more.”

“I’ve heard about this one before, but my doctor is more familiar with the other one.”

“My child is non-ambulatory, so there are no trials for him.” 

These comments are often made by parents of children with Duchenne on social media and at Duchenne events, who are anxious to participate in a trial but need more information on the options that exist. Comments like these are the reason why Jett Foundation began hosting Educational Roundtables in 2013, to ensure that families everywhere-whether they live in Baltimore or New Orleans-have access to information they need to make important decisions that could help their child and advance the Duchenne drug development field forward. 

We invite you to join us at Jett Foundation’s Educational Roundtables in St. Louis on Saturday, May 6th at the Marriott St. Louis Grand Hotel, 10am-2pm. For more information on future planned roundtables in Portland, Oregon on May 20th, San Francisco California on June 3rd, and Denver, Colorado on July 15th, please email danielle@jettfoundation.org.