The Endowed Chair in Pediatric Neuromuscular Disorders and professor of clinical pediatrics and neurology, at Children’s Hospital Colorado discussed the future of SMA treatment.
“We’re not done yet. Just because we have 3 drugs that are available [for patients with spinal muscular atrophy], we’re not complacent about this space.”
The spinal muscular atrophy (SMA) treatment paradigm has shifted drastically in the past few years with the approval of 3 treatments: nusinersen (Spinraza; Biogen), onasemnogene abeparvovec (Zolgensma; Avexis), and risdiplam (Evrysdi; Genentech/Roche). Despite this recent string of successes, though, those who are treating patients and researching therapeutic targets have yet to feel satisfied, according to Julie Parsons, MD.
Parsons, who serves as the primary investigator for the ongoing phase 4 RESPOND trial of nusinersen, is also the Haberfeld Family Endowed Chair in Pediatric Neuromuscular Disorders, and professor of clinical pediatrics and neurology at Children’s Hospital Colorado and the University of Colorado School of Medicine, spoke with NeurologyLive about the current state of the science. She noted the importance of considering rational therapeutic approaches and the collection of more high-quality data as more studies are conducted. She explained just how these goals go hand in hand, as this additional data can be valuable in developing those approaches.
In this conversation, Parsons also explored some of the obstacles that remain in treating the disease and shared her outlook on how the field is expected to continue to advance over the next few years.