Spinraza Stabilizes Motor Function for Adults With SMA, Study Reports
Treatment with Spinraza (nusinersen) modestly improved muscle strength and stabilized motor function for adults with spinal muscular atrophy…
Articles by Marisa Wexler, MS
Parents of SMA Children Put Health as Top Priority at COVID-19’s Onset
For parents, protecting the health and well-being of a child with spinal muscular atrophy (SMA) was a top priority…
Type 1 Children on Evrysdi Continue to Hit Milestones After 3 Years
Children with spinal muscular atrophy (SMA) type 1 who started treatment with Evrysdi (risdiplam) in the first months of…
#AAN2022 – Apitegromab Prompts Motor Improvements in TOPAZ Trial
Treatment with the experimental muscle-directed therapy apitegromab improved motor function for many of the children and young adults with…
Europe Awaits Proposed New Framework for Sharing Health Data
The European Commission is expected to propose a new governing framework for health data next month, called the European Health…
Researchers Share Story of Patient Who Inspired Them
Researchers in Hong Kong have shared a case report about how they were inspired and humbled by a teenage girl…
#AAN2022 – Verana Health Launches Resource to Support SMA Research
Verana Health has launched a service called Neurology Qdata, which contains disease-specific datasets to help facilitate neuroscience research and…
‘Garbage Disposal’ Protein May Help in Predicting Response to Spinraza
In children with spinal muscular atrophy (SMA) who begin treatment with Spinraza (nusinersen) after they are 2 months…
SMA Screening in Latvia Can Diagnose Babies Within 2 Weeks of Birth
A newborn screening program for spinal muscular atrophy (SMA) in Latvia can diagnose babies within two weeks of birth,…
#MDA2022 – SMA Babies Given Zolgensma Early Able to Stand, Walk
Most of the children with spinal muscular atrophy (SMA) who received the gene therapy Zolgensma (onasemnogene abeparvovec) in their…