• Yohei Harada, Vamshi K Rao, Kapil Arya, Nancy L Kuntz, Christine J DiDonato, Galia Napchan-Pomerantz, Amit Agarwal, Vikki Stefans, Masahisa Katsuno, and Aravindhan Veerapandiyan.
• Department of Neurology, University of Arkansas for Medical Sciences, Little Rock, Arkansas, USA.
• Muscle Nerve. 2020 Oct 1; 62 (4): 550-554.

BackgroundData on combining molecular therapies that increase survival motor neuron protein for spinal muscular atrophy type 1 (SMA1) is lacking.MethodsThis was a retrospective study describing our centers' experiences in treating SMA1 patients with combination therapy.ResultsFive children received nusinersen and onasemnogene abeparvovec-xioi (onasemnogene). Four were receiving nusinersen prior to onasemnogene. Nusinersen was continued in three. Marked liver enzyme elevations resulted in prolonged corticosteroid treatment in two patients with hospitalization and liver biopsy in one; milder liver enzyme elevations were noted in the other two. One patient received onasemnogene first, and then nusinersen. No adverse effects were noted. All patients improved.ConclusionsCombination molecular therapy is tolerated in SMA1 patients. Further studies are needed to determine whether there are circumstances in which combination therapy would be more efficacious than either monotherapy. Prolonged corticosteroid use and liver toxicity monitoring may be necessary with onasemnogene therapy.© 2020 Wiley Periodicals LLC.

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