New UBA1 Mutation Linked to Atypical SBMA in Iranian Family
A new mutation in the UBEA1 gene was identified as the cause of an atypical form of spinal and bulbar…
Articles by Marta Figueiredo, PhD
Better Survival Trends Seen With Spinraza in SMA Children in Sweden
The introduction of Spinraza (nusinersen) as a treatment for spinal muscular atrophy (SMA) in Sweden was associated with…
Perceived Physical Fatigue and Fatigability Frequent in SMA
The sense of physical fatigue and perceived fatigability, or the impact of reduced physical performance on daily life activities, are frequent…
Robotic Suit Improves Joint Strength, Mobility in 3 SMA Type 2 Children
Rehabilitation sessions using a robotic suit called ATLAS 2030 improved joint strength and mobility in three boys with spinal…
Initiating Treatment Early Called More Critical Than Therapy Choice
Initiating a disease-modifying therapy (DMT) before the onset of symptoms and at the earliest time possible results in the…
Regular Physical Therapy Linked to Better Results in Spinraza Study
Undergoing physical therapy nearly every day while on Spinraza (nusinersen) leads to significantly greater motor improvements than occasional sessions…
2 Given Spinraza as Newborns Still Showing Normal Development
Two children genetically diagnosed with spinal muscular atrophy (SMA) before birth and starting treatment with Spinraza (nusinersen) within the…
First Patient Enrolled in Phase 3 SMA Trial of Taldefgrobep Alfa
A Phase 3 clinical trial evaluating Biohaven Pharmaceuticals’ taldefgrobep alfa as an add-on therapy in children and young adults with…
Zolgensma Helps Pre-symptomatic Babies Attain Motor Milestones
Zolgensma safely and effectively halts disease progression in infants genetically diagnosed with spinal muscular atrophy (SMA) but not…
New Way of SMA Genetic Screening Faster and Reliable, Study Reports
Researchers in China report having developed a fast and reliable way for a genetic diagnosis of spinal muscular atrophy (SMA)…