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Gene therapy, new drug for rare but deadly disease in kids

Staff Writer |
Babies born with a previously untreatable degenerative nerve disease now have two fresh sources of hope for their future.

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Two innovative new therapies for spinal muscular atrophy (SMA) type 1 have proven highly effective in clinical trials, researchers report.

Babies with SMA are born without the gene that promotes production of survival motor neuron (SMN) protein. Without this protein, nerve cells in the spinal cord and brain stem stop working and start to die off.

These babies slowly lose the ability to move their arms and legs. Those with the most severe form, SMA type 1, eventually lose the ability to breathe on their own and rarely survive beyond 2 years of age.

Two research groups say they've produced breakthrough therapies for these children.

First, a new genetic treatment employed a DNA-loaded virus to replace the missing SMN1 gene with a fresh, healthy copy of the gene.

Second, an already-approved drug called nusinersen (Spinraza) was used to promote production of the crucial nerve protein by a backup gene called SMN2.

Both approaches increased survival in babies with SMA and preserved or improved their motor function, the researchers said.


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