Muscles Controlling Eye Movements Not Affected by SMA, Study Finds
Adults with spinal muscular atrophy (SMA) usually do not experience weakness or impairment of any sort in the muscles…
Articles by Joana Carvalho, PhD
Spinraza Trial Begins Treating Children Who Failed to Respond to Gene Therapy
The first patient has been dosed in a Phase 4 trial exploring the safety and potential benefits of Spinraza (nusinersen)…
Muscle MRI Captures Spinraza’s Effects on Tissue Fibers in Type 3 Brothers
A newer imaging technique called diffusion tensor imaging (DTI) — a type of muscle MRI — is able to identify small…
SMA Type 1 Infants Treated With Zolgensma Achieving Milestones, Data Show
Infants with spinal muscular atrophy (SMA) type 1 who received a single infusion of Zolgensma continue to achieve developmental…
Infants Receiving Evrysdi Continue to Improve and Achieve Motor Milestones, Phase 2/3 Trial Data Show
Infants with spinal muscular atrophy (SMA) type 1 receiving the therapeutic dose of Evrysdi (risdiplam) continue to…
FDA Grants Rare Pediatric Disease Designation to SRK-015 for SMA
The U.S. Food and Drug Administration (FDA) has granted the designation of rare pediatric disease to SRK-015,…
Domain-specific Mutations Affect Stability of SMN Protein in Fruit Fly Model of SMA
Mutations in a specific region of the survival motor neuron (SMN) protein cause it to lose its stability at high…
Imaging Tools to Guide Spinraza’s Use May Help Lower Complications
Adopting image-guided techniques may help physicians lower the risks of complications associated with the administration of Spinraza (nusinersen) in…
European Commission Grants Conditional Approval to Zolgensma for Treating SMA
The European Commission (EC) has granted conditional approval to Zolgensma (onasemnogene abeparvovec) for treating patients with spinal…
Feeding Issues Still Common Among SMA Type 1 Infants, Despite New Therapy Options
Despite recent treatment advances, feeding difficulties are still one of the most common complications for infants with spinal…