The 6-month-old patient died during a Phase 3 European trial testing the gene therapy for spinal muscular atrophy, a muscle-wasting disease. Because the patient died of a respiratory infection followed by neurological complications, an investigator believed the event could be related to the treatment.
The death, which brought up safety concerns about the treatment, also prompted an investigation by the drugmaker.
The investigation and coroner report revealed that the cause of death was brain damage from oxygen deprivation, rather than brain damage from a toxic drug agent. This revealed that the spinal muscular atrophy caused the infection, which lead to oxygen deprivation and caused the death.
The report helps allay concerns about the risks of the $2.1 million-per-patient treatment.
Read the full report here.
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