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New data is presented at MDA meeting of Zolgensma (onasemnogene abeparvovec) from the SMART study for the treatment of spinal muscular atrophy – Novartis

Written by | 11 Mar 2024

Novartis presented new data that continue to support the clinical benefits of Zolgensma (onasemnogene abeparvovec), the only one-time gene therapy for the treatment of spinal muscular atrophy (SMA)…. read more.

Majority of newborn babies with spinal muscular atrophy (SMA) treated with Evrysdi (risdiplam) are able to sit independently after 1 year of treatment – Roche

Written by | 10 Oct 2023

Roche presented positive results from the primary analysis of the ongoing RAINBOWFISH study assessing the efficacy and safety of Evrysdi (risdiplam) in babies with pre-symptomatic SMA (n=26), aged… read more.

European Commission approves Evrysdi for babies under two months old with spinal muscular atrophy (SMA) – Roche

Written by | 5 Sep 2023

Roche announced that the European Commission has approved the extension of the Evrysdi (risdiplam) European Union (EU) marketing authorisation to include infants with a clinical diagnosis of SMA… read more.

Roche presents new data for Evrysdi at World Muscle Society (WMS) 2021 highlighting new advances for people living with rare neuromuscular disorders

Written by | 23 Jan 2022

Roche announced data from across its growing neuromuscular portfolio at the World Muscle Society (WMS) Virtual Congress 20 – 24 September 2021. The presentations included additional results from… read more.

Genentech’s Evrysdi continues to improve motor function and survival in babies with Type 1 Spinal Muscular Atrophy

Written by | 23 Apr 2021

Genentech, announced new 2-year data from Part 2 of FIREFISH, a Phase II/III global study evaluating Evrysdi (risdiplam) in infants aged 1-7 months at enrollment with symptomatic Type… read more.

Evrysdi approved by European Commission as first and only at home treatment for spinal muscular atrophy – Roche

Written by | 15 Apr 2021

Roche announced that the European Commission (EC) has approved Evrysdi (risdiplam) for the treatment of 5q spinal muscular atrophy (SMA) in patients two months of age and older,… read more.

Roche announces results from Evrysdi study in infants with Type 1 spinal muscular atrophy (SMA) published in NEJM

Written by | 9 Mar 2021

Roche announced that the New England Journal of Medicine has published Evrysdi (risdiplam) data from the dose finding Part 1 of the pivotal FIREFISH study in infants with… read more.

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