Evrysdi (risdiplam) is a disease-modifying therapy, taken daily, that’s approved for children and adults with spinal muscular atrophy (SMA). It is the only approved SMA treatment that can be taken orally or via feeding tube, and the first that can be administered at home.
Chugai Pharmaceutical, part of the Roche Group, is asking that risdiplam be approved as a daily oral treatment for spinal muscular atrophy (SMA) patients in Japan. A new drug application seeking approval, submitted to that country’s Ministry of Health, Labour and Welfare (MHLW), may be given priority review…
Evrysdi (risdiplam) is an oral liquid treatment approved in the U.S. for infants through adults with spinal muscular atrophy (SMA) — patients age 2 months and older with any disease type. Roche and Genentech developed Evrysdi in collaboration with PTC Therapeutics and the SMA…
The U.S. Food and Drug Administration (FDA) has approved Evrysdi — formerly known as risdiplam — as the first oral and at-home treatment for adults, children, and infants 2 months of age and older with all types of spinal muscular atrophy (SMA). With this action, Evrysdi — marketed by…
Risdiplam continues to sustain high levels of the SMN protein and improve motor function in children and young adults with spinal muscular atrophy (SMA) types 2 and 3, two-year data from the first part of the SUNFISH trial show. It also safely and effectively raises SMN levels in patients previously treated…
As I lay sideways on the operating table for my most recent Spinraza injection, my neurologist asked me about an upcoming SMA therapy. He and I like to make small talk about medical breakthroughs while a radiologist jams a needle into my neck. We were discussing…
Roche’s experimental oral treatment risdiplam can safely and effectively halt disease progression in infants with type 1 spinal muscular atrophy (SMA) under 7 months of age, according to top-line data from Part 2 of the FIREFISH trial. After one year of treatment, most of these 41 babies…
The systemic nature and high specificity of risdiplam — an oral treatment under review in the U.S. to possibly treat all types of spinal muscular atrophy (SMA) — are key to its effectiveness and favorable safety profile. In an interview with SMA News Today, Paulo Fontoura, MD,…
The U.S. Food and Drug Administration (FDA) asked for the SUNFISH clinical trial’s top-line data that necessitated a delay in its review of whether risdiplam could be an oral treatment for spinal muscular atrophy (SMA), and for good reason — these pivotal results could lead to the treatment being available…
The U.S. Food and Drug Administration (FDA) has extended its review of risdiplam as an oral treatment for spinal muscular atrophy (SMA) by three months, pushing the decision due date to August 24 from May 24. This extension was based on additional clinical data submitted by Roche — one…