Barry J Byrne, MD, PhD, on Making Decisions in the Face of Rapidly Expanding Treatment Options

“The decision process for families is actually quite challenging now because while there are various studies that children can enter into for new therapeutic modalities, there are also a host of approved therapies now that are available and the treatment landscape continues to broaden. We'll see new approaches coming frequently and it's really incumbent upon every provider to remain familiar with all the options that are available to patients and help guide them as to what's best for their child and their family."

Patients with Duchenne muscular dystrophy (DMD) today have the choice between a number of disease-modifying treatment options, such as the several FDA-approved exon-skipping therapies and adeno-associated virus (AAV) vector-based gene therapy in the form of Sarepta Therapeutics’ delandistrogene moxeparvovec-rokl (marketed as Elevidys). Beyond FDA-approved options, patients also have the choice to enroll in one of the many currently ongoing trials testing new investigational drugs, gene-editing approaches, and other experimental therapies. As such, the choice facing patients, families, and their doctors is more complicated than ever for DMD.

CGTLive® recently interviewed Barry J Byrne, MD, PhD, the chief medical advisor of Muscular Dystrophy Association (MDA) and a physician-scientist at the University of Florida, to get his insight on the navigation of this process for the aforementioned groups. Byrne discussed the current landscape of care and how Elevidys has altered that landscape. He also spoke about the work of DMD advocacy groups like MDA and CureDuchenne in distributing important information to patients and families for educational purposes. Byrne additionally discussed several promising investigational treatments that are being evaluated by various companies and institutions right now and pointed out that the potential for combination treatments may expand the breadth of decision-making even further.