Advances in translation research in SMA: Result of clinical trials

October 1st 2018 08:30 - 09:00

Dr. Chiriboga is Professor of Neurology and Pediatrics at Columbia University Medical Center (CUMC) and director of Pediatric MDA Care Center at CUMC. In her capacity as member of the SMA Clinical Research Center of Columbia University, she has participated in translational SMA research, including the early open label and later sham controlled studies with nusinersen in infants and older onset SMA patients. Currently she participates in the nusinersen extension study (CS11), and phase 2/3 trials with small molecule RG7916 and gene replacement therapy. As a clinician, she also treats SMA patients with commercial nusinersen.


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