The purpose of SMA Europe is to provide a framework to stimulate collaboration and accelerate translational research pathways in SMA and promote patient care.

  1. Novartis provides update on LMI070 and its clinical development

    stethoscopeNovartis provides update on LMI070, a potential treatment for SMA Type 1, through the statement below:

    “We have made the difficult decision to pause enrollment for our study of LMI070 for the treatment of Type 1 Spinal Muscular Atrophy (SMA). Animal safety studies were taking place in parallel with this trial, which is a common practice when studying potentially life threatening diseases to help bring medicines to patients faster. Results from an animal study, using daily dosing for a year compared to weekly dosing in the human study, showed unexpected injuries to the peripheral nerves and spinal cord, testes, and blood vessels in the kidney.

    Patient safety is paramount to us and after a review of these results and in consultation with our Data Monitoring Committee, a team of independent experts who monitor the trial’s safety results, we decided to continue the trial but pause additional enrollment while we do further analysis.

    All patients currently enrolled in the trial are being closely monitored and the teams at the clinical trial sites are working with their families to explain the findings, answer their questions, and help them decide whether they would like to continue. We are also working closely with our teams and health authorities to determine appropriate next steps.”

    LMI070 is an orally available drug, which works by increasing the amount of functional SMN protein produced by the “back-up” gene, SMN2, through modifying its splicing. Please see the clinical trials section of our website for more information on how this compound works.

    For more information on this trial, please see the ClinicalTrials.Gov website.