The Muscular Dystrophy Association‘s (MDA) annual Clinical & Scientific Conference kicked off over the weekend, bringing together patients,…
Articles by Marisa Wexler, MS
SMA Europe is funding five new research projects that are intended to advance understanding of spinal muscular atrophy…
The mother of a baby with spinal muscular atrophy (SMA) was given Evrysdi (risdiplam) for the last few…
While effective treatment is lengthening life and improving its quality, children with spinal muscular atrophy (SMA) type 1 often…
The U.S. Food and Drug Administration (FDA) has approved a new tablet formulation of Evrysdi (risdiplam), an approved oral treatment…
Cure SMA is leading an advocacy campaign for new research funding from the U.S. Department of Defense (DOD) that…
Scholar Rock is asking the U.S. Food and Drug Administration (FDA) to approve apitegromab, its experimental add-on therapy…
An algorithm that takes into account clinical measures of motor symptom severity alongside data on factors like age and height…
SMA Europe is launching an initiative to share personal stories of how treatment affects the lives of people with…
The activity of many genes may be dysregulated in spinal muscular atrophy (SMA), but the way the disease affects…