Author Archives: Marisa Wexler MS

Risdiplam, Oral SMA Treatment, Up for Approval in Japan

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…

Disparities Found in Parents, Children’s Perceptions of SMA

Perceptions about illness often don’t line up between children with spinal muscular atrophy (SMA) and their parents, with mothers and fathers often perceiving disease severity as worse than their children do, according to a recent study. Importantly, children’s perceptions of their disease are closely associated with their quality of…

Cure SMA Details Its ‘Clinical Trial Readiness Program’

A readiness program to help clinical trial sites prepare to run spinal muscular atrophy (SMA) studies has been established by Cure SMA in collaboration with the pharmaceutical industry, other patient advocacy groups, and research institutes. The program is described in a position statement, “The SMA Clinical…