Tag: spinal muscular atrophy

1. In this single-group trial, among infants predicted to develop type 1 spinal muscular atrophy (SMA) who were treated presymptomatically ...

1. Risdiplam treatment increased the survival of motor neuron protein in the blood of infant patients diagnosed with type 1 ...

1. Intrathecal injections of nusinersen in pediatric later-onset spinal muscular atrophy (SMA) patients were associated with increased clinical motor function ...