Safety and tolerability of RG7800 in healthy adults and patients with spinal muscular atrophy

Takeaway

  • In Phase 1 studies, the oral splicing modifier, RG7800 was well-tolerated in healthy subjects and patients with spinal muscular atrophy (SMA), and Survival of Motor Neuron (SMN) protein levels were increased in the blood of patients with SMA.

Why this matters

  • Oral therapies that can modify SMN protein levels are a research priority as a potential treatment for spinal muscular atrophy SMA.