#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…
Articles by Marisa Wexler, MS
‘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…
#MDA2022 – Newborns Treated With Evrysdi Standing, Walking 1 Year Later
In the RAINBOWFISH trial testing oral Evrysdi (risdiplam) in babies with spinal muscular atrophy (SMA) but no evident symptoms, most…
#MDA2022 – Data: Zolgensma Given After 6 Months Benefits SMA Babies
Even when given to young children with spinal muscular atrophy (SMA) at ages older than 6 months, the gene…
#MDA2022 – Evrysdi Safe at 1 Year in Previously Treated Patients
Treatment with Evrysdi (risdiplam) continues to be safe and well tolerated in people with spinal muscular atrophy (SMA) who…
#MDA2022 – Motor Benefits of Evrysdi for Children, Adults in SUNFISH Now at 3 Years
Children and young adults with spinal muscular atrophy (SMA) types 2 and 3 continue to report improved or stabilized…
#MDA2022 – ‘Amazing’ Walking Ability Seen in SMA Children on Spinraza
Most children with spinal muscular atrophy (SMA) who were presymptomatic and treated with Spinraza (nusinersen) in the NURTURE…
Newborn Screening for SMA Now Launched in Canadian Province
Babies born in the Canadian province of Saskatchewan will now be tested for spinal muscular atrophy (SMA) as part…