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Spinal Muscular Atrophy clinical trials at UC Davis
1 research study open to eligible people

  • A Study of the Experimental Medicine LMI070 (Branaplam) in Infants With Type 1 Spinal Muscular Atrophy (muscle weakness in spine)

    open to all eligible people

    An open-label, multi-part, first-in-human study of oral branaplam in infants with Type 1 spinal muscular atrophy. The purpose of this study is to evaluate the safety, tolerability, pharmacokinetics (PK), pharmacodynamics (PD) and efficacy after 13 weeks; and to estimate the Maximum Tolerated Dose (MTD) of orally administered branaplam; and to identify the dose that is safe for long term use as well as that can provide durable efficacy optimal dosing regimen in patients with Type 1 SMA.

    Ekaterinburg, and other locations

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