Julie Parsons, MD

John Brandsema, MD, and Julie Parsons, MD, review the evolution of combination therapy in clinical practice for the management of SMA.

John Brandsema, MD, comments on the importance of patient compliance to therapy to manage spinal muscular atrophy.

Experts in pediatric neurology share insights on the role of small molecule treatment for SMA through the recent FDA approval of risdiplam and discuss factors to consider when tailoring therapy for individual patients.

John Brandsema, MD, leads the discussion on the concept of gene transfer for the management of SMA and the recent approval of onasemnogene abeparvovec-xioi.

Experts in pediatric neurology provide insight on the efficacy of nusinersen and treatment response in patients with SMA.

John Brandsema, MD, and Julie Parsons, MD, discuss the approval of nusinersen for the treatment of spinal muscular atrophy and their personal experiences regarding drug safety and administration in patients.

Expert pediatric neurologists review the importance of early recognition and referral to neurologists for the diagnosis of spinal muscular atrophy and when it’s appropriate to approach treatment.

Julie Parsons, MD, comments on the role of primary care providers and gene testing for the early diagnosis of spinal muscular atrophy.

Identifying spinal muscular atrophy through early diagnosis using prenatal and newborn screenings is discussed.

Julie Parsons, MD, discusses the importance of a multidisciplinary team approach to managing SMA in clinical practice.

John Brandsema, MD, leads the discussion on the 5 main types of spinal muscular atrophy and the evolution of disease classification.

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.

The primary investigator of the phase 4 RESPOND study discussed the role that postmarketing studies can play in assessing treatments for SMA.

The primary investigator of RESPOND discussed the trial’s goals and measures, as well as insight into why clinical trials like it are important in spinal muscular atrophy.