This Plain Language Summary of Publication article (PLSP) from Expert Review of Neurotherapeutics explains spinal muscular atrophy (SMA), a rare genetic disease where low SMN protein levels cause motor neurons to die, leading to muscle weakness and loss of movement. While current SMN-targeted treatments help by increasing SMN protein, many patients still have reduced motor function. The SAPPHIRE trial tested apitegromab, a new treatment that targets myostatin in muscles of SMA patients already receiving SMN-targeted therapies to see if it could further improve motor function.

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This PLSP is based on an article called ‘Safety and efficacy of apitegromab in nonambulatory type 2 or type 3 spinal muscular atrophy (SAPPHIRE): a phase 3, double-blind, randomised, placebo-controlled trial’ and was published in The Lancet Neurology.

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