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FDA approves $2.1m gene therapy for infants with rare genetic disease

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Bill Davis
May 24, 2019 20:51
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The Food and Drug Administration (FDA) has issued its first approval for a gene therapy treatment targeting young children.

The gene therapy is called Zolgensma, and it is designed to treat the rare genetic disease spinal muscular atrophy in children under the age of two.

The FDA’s Acting Commissioner Ned Sharpless called the approval a ‘milestone’ for gene therapy.

Spinal muscular atrophy (SMA) is the rare result of a genetic mutation involving the survival motor neuron 1 gene.

In the most common (and severe) cases, SMA onset starts during infancy, with symptoms appearing by the age of only six months old.

The motor neuron problems caused by SMA result in severe complications, including trouble breathing and swallowing.

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Bill Davis
May 24, 2019 20:51
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