On Sunday, Jesy Nelson, a former member of beloved girl group Little Mix, shared the emotional news that her eight-month-old twin daughters, Ocean Jade and Story Monroe, had been diagnosed with Spinal ...

Biomarkers for SMA were identified using LC-MS and machine learning, revealing differences in protein expression across SMA types. CNTN1 and NRXN3 proteins showed potential as prognostic biomarkers, ...

Although a profound transformation has occurred in both survival and clinical course, long-term data on the efficacy and safety of disease-modifying therapies is required to inform their use in ...

A multivariable analysis suggested the safety and efficacy of onasemnogene abeparvovec (OA) in the treatment of older and heavier children with spinal muscular atrophy (SMA) type 1. No serious ...