Trial offers hope of a treatment for spinal muscular atrophy
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.