26 newborns diagnosed with SMA in Russia through pilot program
Twenty-six children were diagnosed with spinal muscular atrophy (SMA) in Russia through a 2022 pilot program, out of nearly…
Articles by Marisa Wexler, MS
Brain fluid biomarkers may help distinguish SMA types, study finds
Levels of certain molecules in cerebrospinal fluid (CSF) — the liquid that surrounds the brain and spinal cord — may…
MScanFit shows promise as novel SMA monitoring tool in study
A novel monitoring tool called MScanFit may be a reliable way to assess the health of nerve and muscle cells…
Study examines side effects observed with SMA gene therapy
Serious side effects that can cause or prolong hospital stays occurred frequently among children with spinal muscular atrophy (SMA)…
Rare sequence variant mutations are cause of 6 SMA cases
Six unusual cases of children with spinal muscular atrophy (SMA) caused by sequence variants, rather than the more common…
Rare C-shaped curve in lower spine seen in boy with SMA type 3
The case of a boy with spinal muscular atrophy (SMA) who had severe hyperlordosis — when the lower part…
Most SMA patients with four SMN2 copies show symptoms in childhood
Without treatment, most people with spinal muscular atrophy (SMA) who have four copies of the “backup” SMN2 gene will…
MDA 2024: Spinraza may benefit some SMA children after Zolgensma
In children with spinal muscular atrophy (SMA) who have a less-than-optimal response to the gene therapy Zolgensma…
MDA 2024: Zolgensma gene therapy safe in children up to 21 kg
The one-time gene therapy Zolgensma (onasemnogene abeparvovec-xioi) didn’t cause any unexpected safety issues among children with spinal muscular…
Children with SMA express good quality of life in small study
Children with spinal muscular atrophy (SMA), even those with substantial physical disability, tend to report that their quality of…