Trial offers hope of a treatment for spinal muscular atrophy

Posted by on September 20, 2016 7:45 pm
Categories: health

Spinal muscular atrophy (SMA) is a leading genetic cause of child death. Existing proposed treatments require injection direct into the spine, a barrier to widespread use. Pip6a-PMO can be injected into a vein – a simpler and safer method – report investigators. In SMA-affected mice, a single dose of Pip6a-PMO extended life from 12 to 167 days – fourteen times longer. A second dose increased that to 457 days.

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