Blood NfL Levels May Mark SMA Severity, Therapy Efficacy in Very Young
Blood levels of neurofilament light chain (NfL) — a marker of nerve cell damage — in children and adolescents with spinal muscular atrophy (SMA) are significantly higher than those of age-matched healthy peers and associate with disease severity, a study found. NfL...
2 SMN2-targeting Therapies Work Better Than 1 in Mouse Model
Treatment with two distinct molecules targeting the “backup” SMN2 gene resulted in higher levels of SMN — the protein lacking in spinal muscular atrophy (SMA) — and longer survival in a mouse model of type 1 disease relative to the use of a single therapy. Notably,...
Most Parents Surveyed in Japan Support SMA Newborn Screening
Despite being mostly unaware of spinal muscular atrophy (SMA), most Japanese parents support newborn screening for the disease, according to data from a small survey-based study. The most cited reasons behind this support included the importance of early diagnosis and...
SMA Type 1 Affects Sensory Nerves as Children Age, Small Study Shows
Sensory nerves — those responsible for transmitting sensory information — are progressively damaged in children with spinal muscular atrophy (SMA) type 1 as these patients age, according to a small study in Italy. Importantly, major functional deficits in the sensory...HCP — SMA News Today
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