Twin girls with type 0 spinal muscular atrophy (SMA) — the most severe form of the disease, which usually…
Articles by Marisa Wexler, MS
Children and adolescents in New Zealand with spinal muscular atrophy (SMA) will be able to access Evrysdi (risdiplan)…
The one-time gene therapy Zolgensma (onasemnogene abeparvovec-xioi) can improve motor outcomes for children with spinal muscular atrophy (SMA)…
A group of proteins called the SNARE complex, which normally helps regulate how nerve cells receive chemical messages from other…
Children with spinal muscular atrophy (SMA) given Zolgensma (onasemnogene abeparvovec-xioi) gene therapy in clinical trials are maintaining, years…
More than 1 in 4 ambulatory spinal muscular atrophy (SMA) patients experienced clinically meaningful improvements in walking ability after being…
Most children with spinal muscular atrophy (SMA) given the gene therapy Zolgensma (onasemnogene abeparvovec-xioi) retain motor function that…
Some videos on YouTube, particularly in-depth ones made by healthcare professionals, provide high-quality and reliable information about treatments for…
In people with spinal muscular atrophy (SMA) who have been on long-term treatment with Spinraza (nusinersen), levels of…
A new computational tool developed by Pacific Biosciences can identify genetic mutations that cause spinal muscular atrophy (SMA)…