Motor gains seen with Zolgensma, especially when given early
The one-time gene therapy Zolgensma (onasemnogene abeparvovec-xioi) can improve motor outcomes for children with spinal muscular atrophy (SMA)…
Articles by Marisa Wexler, MS
Study sheds light on how SMN protein deficiency can cause SMA
A group of proteins called the SNARE complex, which normally helps regulate how nerve cells receive chemical messages from other…
MDA 2023: Long-term motor gains seen for SMA children on Zolgensma
Children with spinal muscular atrophy (SMA) given Zolgensma (onasemnogene abeparvovec-xioi) gene therapy in clinical trials are maintaining, years…
Ambulatory SMA patients on Spinraza show walking gains
More than 1 in 4 ambulatory spinal muscular atrophy (SMA) patients experienced clinically meaningful improvements in walking ability after being…
Better motor function seen in SMA children given Zolgensma: Analysis
Most children with spinal muscular atrophy (SMA) given the gene therapy Zolgensma (onasemnogene abeparvovec-xioi) retain motor function that…
SMA treatment videos on YouTube vary as source of solid information
Some videos on YouTube, particularly in-depth ones made by healthcare professionals, provide high-quality and reliable information about treatments for…
Tau Protein May Be Marker of Long-term Response to Spinraza
In people with spinal muscular atrophy (SMA) who have been on long-term treatment with Spinraza (nusinersen), levels of…
New Genetic Analysis Method Aids SMA ‘Silent Carrier’ Detection
A new computational tool developed by Pacific Biosciences can identify genetic mutations that cause spinal muscular atrophy (SMA)…
Diagnosis Through Newborn Screening Yields Better Outcomes
Newborn screening for spinal muscular atrophy (SMA), coupled with early access to disease-modifying therapies, can improve the likelihood…
Muscle Strengthener Found to Ease Patient Fatigue in Small Study
Treatment with the muscle strengthener pyridostigmine may reduce patient-reported fatigue among people with spinal muscular atrophy (SMA), results…