Pooled Safety Data from the risdiplam▼ clinical trial development program

FIREFISH (NCT02913482) is assessing the safety, tolerability, PK/PD and efficacy of risdiplam▼ in infants with Type 1 SMA aged 1–7 months. SUNFISH (NCT02908685) is assessing the safety, tolerability, PK/PD and efficacy of risdiplam in patients with Type 2 or 3 SMA aged 2–25 years. JEWELFISH (NCT03032172) is assessing the safety, tolerability and PK/PD of risdiplam in in non-naïve patients with SMA, aged 6 months to 60 years. This presentation reports updated pooled safety analyses from FIREFISH Parts 1 and 2, SUNFISH Parts 1 and 2 and JEWELFISH. This analysis will add to the understanding of the longer-term safety profile of risdiplam in individuals with SMA.

{{assetTitle}}

Authors {{assetAuthors}}

Published date{{formattedPublishDate}}

{{assetDescription}}

Welcome to Medically

The Roche Science Hub

This website is a non-promotional global resource intended to facilitate transparent scientific exchange regarding developments in medical research, diagnostics, and disease management. It is intended for healthcare professionals.

I am a healthcare professional

Not a healthcare professional? Browse:

Roche's patients website Roche.com

This global website is intended for healthcare professionals outside the UK, US, Canada and Australia. The content on this website may include scientific information about experimental or investigational compounds, indications and services that are not approved or valid in your jurisdiction. Registration status and prescribing information of medicinal products may differ between countries. Please refer to local product information for any medicinal products mentioned on this website. Information available on this website does not constitute professional medical advice, and Roche and Genentech accept no responsibility for access to or use of the same.

You are Leaving Medically

By following this link, you are leaving Roche Website and entering a site that is not owned or controlled by Roche. Roche does not take any responsibility for acces to or use of this website, nor for any content therein.

Leave Site Stay on Medically