Combination molecular therapies for type 1 spinal muscular atrophy

Yohei Harada, Vamshi K. Rao, Kapil Arya, Nancy L. Kuntz, Christine J. DiDonato, Galia Napchan-Pomerantz, Amit Agarwal, Vikki Stefans, Masahisa Katsuno, Aravindhan Veerapandiyan*

*Corresponding author for this work

Research output: Contribution to journalArticlepeer-review

41 Scopus citations


Background: Data on combining molecular therapies that increase survival motor neuron protein for spinal muscular atrophy type 1 (SMA1) is lacking. Methods: This was a retrospective study describing our centers' experiences in treating SMA1 patients with combination therapy. Results: Five 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. Conclusions: Combination 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.

Original languageEnglish (US)
Pages (from-to)550-554
Number of pages5
JournalMuscle and Nerve
Issue number4
StatePublished - Oct 1 2020


  • SMA
  • combine
  • gene therapy
  • nusinersen
  • onasemnogene

ASJC Scopus subject areas

  • Clinical Neurology
  • Physiology (medical)
  • Cellular and Molecular Neuroscience
  • Physiology


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