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.

[caption id="attachment_5125" align="alignnone" width="300"]<img class="wp-image-5125 size-medium" src="https://microbiozindia.com/wp-content/uploads/2020/04/450px-Polio_spinal_diagram-en.svg_-300x219.png" alt="" width="300" height="219" /> Location of neurons affected by spinal muscular atrophy in the spinal cord[/caption]

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.

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