Sharon Hesterlee, PhD, on What Attendees Can Look Forward to at MDA’s 2024 Conference
The chief research officer of the Muscular Dystrophy Association also went over the highlights from last year’s meeting.
“I think it's a really exciting time right now in the world of neuromuscular disease. We've really entered the treatment era with a vengeance after many, many years of not having effective treatments for these diseases.”
The Muscular Dystrophy Association (MDA) Clinical & Scientific Conference is an annual event that brings together both practicing clinicians and medical researchers under one roof to discuss the treatment of neuromuscular diseases and the ongoing development of new therapeutics for these disorders. The 2024 meeting is set to be held on March 3-6, 2024, in Orlando, Florida. CGTLive™ extensively covered the 2023 meeting and will continue this tradition in 2024.
In October 2023, as part of the lead up to the 2024 conference, CGTLive sat down with Sharon Hesterlee, PhD, the chief research officer of MDA, to discuss the upcoming meeting. Hesterlee gave an overview of what the conference is and what makes MDA’s annual meetings unique among medical conferences, emphasizing the focus on both clinical care and basic research. She also spoke about some of the highlights from the 2023 conference, specifically noting the keynote speech delivered by Peter Marks, MD, PhD, the director of the Center for Biologics Evaluation and Research at the FDA, and discussions surrounding the then-pending FDA decision on Sarepta Therapeutics’ delandistrogene moxeparvovec (SRP-9001, now marketed under the name Elevidys), which was ultimately approved by the FDA for a Duchenne muscular dystrophy indication on on June 22, 2023.
Hesterlee also spoke about some things that attendees of the 2024 meeting can look forward to. In particular, she noted a series on practical considerations for gene therapies; the Lab to Life Series, which will focus on the development of therapies from research on disease mechanisms as a starting point; a new live poster tour; and a new system of tagging posters and presentations by disease group in order to make following topics of interest at the conference more straightforward.
Click here to register for the upcoming 2024 MDA Conference.
Considerations for Administering Hemoglobinopathy Gene Therapies From a Pharmacist’s Perspective
April 20th 2024Alexis Kuhn, PharmD, BCOP, a pediatric oncology pharmacist at Mayo Clinic, discussed the incorporation of the recently FDA-approved gene therapies for SCD and TDT into the work of pharmacists.
