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Study suggests new strategy for spinal muscular atrophy

Some little souls are here with us for just a few months, but leave lasting impressions. A Nature Biotechnology article this week gives yet another bit of hope for families whose babies have spinal muscular atrophy (SMA), one of those rare diseases you probably never heard of.

I'd heard of it because a family in my town had a girl born with the disease who never made it to her first birthday. Then my mom got Lou Gehrig's disease, and, as I worked with her and then with the ALS Association, the efforts to address SMA sounded more familiar - both diseases destroy the motor neurons, which allow us to move. So now, in mice who closely match the human disease, researchers have shown that a particular gene therapy sustains life beyond what is expected, and beyond what was achieved before. Its typical for test therapies to add a few weeks in both Lou Gehrig's and in SMA. But this time, Brian Kaspar, PhD, who has worked on both diseases, batted out of the park, adding over 200 days to these small mammals normally short lifespans.

Mixing the two diseases as I've done is intentional. Anyone who has watched either disease sees that there is something similar going on. Years ago, while observing at the Stanford neonatal intensive care unit, I heard about afflicted babies. Now, back at work reporting at Stanford again, one of the first things I am thinking about is SMA - and am glad to hear that researchers here are also hard at work on this problem. A trial continues to test a therapy. One day, maybe both of these problems will be helped.

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