Roche: presents positive results for SMA
(CercleFinance.com) - Roche today presented positive results from the primary analysis of the ongoing RAINBOWFISH study evaluating the efficacy and safety of Evrysdi (risdiplam) in babies with pre-symptomatic SMA (n = 26), aged from birth to six weeks.
The data was presented at the 28th World Muscle Society (WMS) Congress, on 3-7 October 2023.
RAINBOWFISH was the first trial to assess cognition using a standardized scale (BSID) as an exploratory endpoint, and results showed cognitive skills typical of normal child development after 1 year of Evrysdi treatment, as assessed by BSID-III.
Evrysdi is the only non-invasive treatment for AMS and can be used within hours of birth, potentially enabling these babies to sit, stand and walk, just like healthy individuals, Roche said.
Evrysdi has now demonstrated its safety and efficacy in babies, children and adults, and this compelling data continues to strengthen the group's confidence in this treatment, it added.
Copyright (c) 2023 CercleFinance.com. All rights reserved.
The data was presented at the 28th World Muscle Society (WMS) Congress, on 3-7 October 2023.
RAINBOWFISH was the first trial to assess cognition using a standardized scale (BSID) as an exploratory endpoint, and results showed cognitive skills typical of normal child development after 1 year of Evrysdi treatment, as assessed by BSID-III.
Evrysdi is the only non-invasive treatment for AMS and can be used within hours of birth, potentially enabling these babies to sit, stand and walk, just like healthy individuals, Roche said.
Evrysdi has now demonstrated its safety and efficacy in babies, children and adults, and this compelling data continues to strengthen the group's confidence in this treatment, it added.
Copyright (c) 2023 CercleFinance.com. All rights reserved.