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Zolgensma gene therapy liver damage usually resolves in months

Liver damage may occur as a side effect of the spinal muscular atrophy (SMA) gene therapy Zolgensma (onasemnogene abeparvovec-xioi), but it usually resolves within a few months of treatment, a study showed. The researchers also found that simultaneous treatment with Zolgensma and Spinraza (nusinersen) does not seem to…

HapSMA test can help ID genetic changes affecting SMA: Study

Testing patient DNA with advanced methods like HapSMA — which can “[analyze] … SMN and its surrounding genes” — can reveal otherwise undetectable genetic changes, such as gene conversion, that affect how severe spinal muscular atrophy (SMA) is, according to a study by researchers in the Netherlands. These novel…

DMTs boost fine motor skills in young SMA patients able to sit

A year of disease-modifying therapies (DMTs) improved motor function, particularly fine motor skills, in people with spinal muscular atrophy (SMA) who could sit, but not walk due to disease progression before they had access to treatment, a study reports. These functional improvements were independent of treatment-related gains in…