Zolgensma® First Gene Therapy for Spinal Muscular Atrophy (SMA)

Dr. Meredith Schultz with AveXis discusses the FDA approval of Zolgensma (onasemnogene abeparvovec-xioi), the first gene therapy for the treatment of spinal muscular atrophy (SMA) in pediatric patients less than 2 yrs of age.  It is given as a one-time infusion into the vein. Zolgensma was not evaluated in patients with advanced SMA.

Dr. Meredith Schultz is a pediatric neurologist specializing in neurogenetic disorders and gene therapy clinical trials.  She is currently the Medical Director of Translational Medicine at AveXis., Inc. Prior to joining AveXis in February 2019, Dr. Schultz directed the pediatric Neuromuscular Program and co-directed the Neurogenetics Program at the University of Wisconsin (UW), Madison. During her time at UW, she cared for many patients with Spinal Muscular Atrophy (SMA) as well as other neuromuscular and neurogenetic disorders.  In addition, she served as the principal investigator for several gene therapies clinical trials for SMA and had the opportunity to treat 6 patients with Zolgensma gene therapy. In addition, she helped to initiate AveXis’sManaged Access Program (MAP) and dosed the first patient in this program in October 2018. After joining AveXis, she has continued to support and be a thought-leader for the MAP program. Her current role in Translational Medicine is to successfully bring gene therapies for neurogenetic disorders from the lab to the patient.  This includes program development for Rett Syndrome– a rare, devastating neuro-developmental disorder affecting primarily young girls, as well as other future gene therapy programs.

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