Novartis to Investigate Second Death in Gene Therapy Trials 

May 2, 2019

Despite positive interim trial results for its experimental gene therapy for spinal muscular atrophy in infants, Novartis announced it will investigate a second trial death that may be related to the same treatment.

The gene therapy, Zolgensma, returned encouraging interim results for 22 babies with Type 1 SMA, the most serious form of spinal muscular atrophy.

An initial death in the trial was found to be unrelated to the treatment, but the second fatality—in a separate European trial—“was deemed possibly related to treatment by the investigator,” the company says.

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