Baby’s Death in Spinal Muscular Atrophy Gene Therapy Trial Could be Linked to Treatment, Company Says

According to a story from BNN Bloomberg, Novartis AG, a drug company that is nearing the completion of the development of a potentially groundbreaking new gene therapy for spinal muscular atrophy, recently released a statement that contained some bad news: The death of an infant in a recent trial of the therapy, which is called Zolgensma, may have been a direct result of treatment. While this has yet to be confirmed, it could cast serious doubt on the safety of the gene therapy.

About Spinal Muscular Atrophy (SMA)

Spinal muscular atrophy is a type of neuromuscular disorder in which the motor neurons are destroyed, leading to muscle wasting. In many cases, the disease is lethal. This disorder is linked to genetic defects of the SMN1 gene. This gene encodes a protein called SMN, and when not present in certain amounts, neurons are unable to function. There are different kinds of spinal muscular atrophy that are categorized by when symptoms first appear. These symptoms may include loss of reflexes, muscle weakness and poor muscle tone, problems with feeding and swallowing, developmental delays, respiratory muscle weakness, tongue twitching, and a bell shaped torso. There are a variety of management strategies for spinal muscular atrophy, but it is still usually fatal in its most severe forms. To learn more about spinal muscular atrophy, click here.

Is Zolgensma Safe?

The results of an autopsy of the six month old infant have yet to be released. The patient died as the result of a severe respiratory infection which was then followed by neurological abnormalities. The event is of special concern as Zolgensma is actually approaching the end of its development process. Novartis is hoping to introduce the gene therapy onto the market in 2019. In fact, an approval decision on Zolgensma from the US Food and Drug Administration (FDA), is only about a month away.

While many of the latest trials of Zolgensma have displayed positive results, another recent death, this time of respiratory failure, signifies another worrying event in the drug testing process. However, this death was not determined to be directly caused by treatment with Zolgensma. 

Unfortunately, only further study and the release of autopsy details will fully reveal the implications of these undesirable incidents.


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