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  1. Article

    Open Access

    Response to: Alfred Sandrock, Wildon Farwell. Letter to the Editor, Comparisons Between Separately Conducted Clinical Trials: Letter to the Editor Regarding Dabbous O, Maru B, Jansen JP, Lorenzi M, Cloutier M, Guérin A, et al. Adv Ther (2019) 36(5):1164-76. doi:10.1007/s12325-019-00923-8

    Omar Dabbous, Benit Maru, Jeroen P. Jansen, Maria Lorenzi in Advances in Therapy (2019)

  2. Article

    Open Access

    Survival, Motor Function, and Motor Milestones: Comparison of AVXS-101 Relative to Nusinersen for the Treatment of Infants with Spinal Muscular Atrophy Type 1

    Infants with spinal muscular atrophy (SMA) type 1 typically face a decline in motor function and a severely shortened life expectancy. Clinical trials for SMA type 1 therapies, onasemnogene abeparvovec (AVXS-1...

    Omar Dabbous, Benit Maru, Jeroen P. Jansen, Maria Lorenzi in Advances in Therapy (2019)