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.
- Michelle Avery, Ph.D from Summit, presented on Summit’s utrophin modulatin program and the clinical trial they are enrolling to study ezutromid in ambulatory boys with Duchenne.
- Randy Perrin from Sarepta, discussed several exon-skipping trials that Sarepta is currently enrolling, for ambulatory patients amenable to the skipping of exons 45, 53, and 51.
- Todd Bazemore from Santhera explained the clinical trial to study idebedone and its effect in delaying the loss of respiratory function in patients on glucocorticosteroids over the age of 10.
- Wendy Eggleston and Hiro Osaki from NSPharma, presented on their clinical trial studying their exon 53 skipping agent in ambulatory patients with Duchenne.
- 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.
- Angelika Fretzen, PhD from Catabasis discussed their MoveDMD trial. A phase I/II trial that is currently active and studying the effects of edasalonexent on Duchenne patients.
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 firstname.lastname@example.org.