Spinal muscular atrophy is no longer a leading cause of inherited infant death in the United
States. Since 2016, three genetic therapies have been approved for the treatment of spinal …

Nusinersen (Spinraza®) improves survival of infants with hereditary proximal spinal
muscular atrophy and motor function in children up to 12 years. Population-based …

Introduction The clinical application of disease modifying therapies has dramatically
changed the paradigm of the management of people with spinal muscular atrophy (SMA) …

Background: Intrathecal administration of nusinersen in adult spinal muscular atrophy (SMA)
patients presents challenges owing to severe scoliosis and previous spinal surgery with …

Background: Spinal muscular atrophy (SMA) is a genetic disorder that leads to progressive
tetraparesis. Nusinersen is the first approved drug for the treatment of SMA and is …

Background and purpose The purpose was to report the results of ultrasound‐guided
lumbar puncture for the administration of nusinersen in spinal muscular atrophy (SMA) …

Background As the first gene therapy for spinal muscular atrophy (SMA), nusinersen is
supposed to be administrated via intrathecal injection regularly for a lifetime. However, for …

The approval of nusinersen for the treatment of spinal muscular atrophy (SMA) has
significantly changed the natural history of the disease. Nevertheless, scoliosis secondary to …

Background Nusinersen is the only approved treatment for all spinal muscular atrophy
(SMA) subtypes and is delivered intrathecally. Distorted spinal anatomy and instrumentation …

Spinal muscular atrophy (SMA) is a hereditary neuromuscular disorder, typically caused by
survival motor neuron 1 (SMN1) gene deletion in chromosome 5q resulting in loss of SMN …