A Rational Approach to Assessing SMA Therapies: Julie Parsons, MD


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

“Nusinersen was approved just 4 years ago. The fact that this changing landscape has just continued to rapidly evolve, and we have more questions now than we did at the beginning when we had no therapies is absolutely fascinating.”

Just this month, Biogen announced that the first patient was treated in its global phase 4 RESPOND study (NCT04488133) of nusinersen (Spinraza) in infants and children with spinal muscular atrophy (SMA). The trial will evaluate the gene therapy’s performance in those who have unmet clinical needs despite treatment with another FDA-approved medicine, Zolgensma (onasemnogene abeparvovec; Avexis).

RESPOND primary investigator Julie Parsons, MD, Haberfeld Family Endowed Chair in Pediatric Neuromuscular Disorders, professor of clinical pediatrics and neurology, at Children’s Hospital Colorado and the University of Colorado School of Medicine, told NeurologyLive that this trial will feature a number of new outcome measures, including assessments of biomarker levels, such as neurofilament.

Parsons shared her thoughts on what information these evaluations will provide for patients with SMA and their physicians, and how they can impact clinical care. As well, she discussed the role that postmarketing studies can play in the attempts to recognize patients who may respond poorly to treatment as the field of medicine continues to strive for precision, personalized treatment.

Biogen announces first patient treated in respond study evaluating benefit of spinraza® (Nusinersen) In patients treated with zolgensma® (Onasemnogene abeparvovec). January 8, 2021. Accessed January 26, 2021. https://investors.biogen.com/news-releases/news-release-details/biogen-announces-first-patient-treated-respond-study-evaluating
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