Roche says risdiplam viable in spinal muscular atrophy preliminary

Overview

• Post By : Kumar Jeetendra


• Source: Reuters


• Date: 28 Apr,2020

Roche Holding AG’s risdiplam showed significant improvement in survival and motor milestones in a clinical trial for infants with type 1 spinal muscular atrophy (SMA), the Swiss drugmaker said on Tuesday.

Location of neurons affected by spinal muscular atrophy in the spinal cord

The Firefish part 2 study met its primary endpoint by demonstrating a significant increase in motor milestones in infants aged 1-7 months after 12 months of treatment, it said.
Safety was consistent with the safety profile observed to date and no new safety signals were identified, it added.