†These authors contributed equally.
Academic Editor: Amedeo Amedei
Introduction: Spinal muscular atrophy (SMA) is a progressive
neurological disease with autosomal recessive transmission that affects motor
neurons, causing their loss and resulting in muscle waste and motor deficiency.
Nusinersen, the first SMN2 pre-mRNA targeted therapy approved by the Food and
Drug Administration and the European Medicines Agency, has demonstrated high
efficacy in improving motor function, as well as respiratory and nutritional
statuses. Materials and Methods: We observed 55 patients
(children/adolescents) diagnosed with spinal muscular atrophy (SMA), who received
nusinersen therapy. To investigate the benefits of physical therapy on
rehabilitation outcomes, we compared the motor evolution of patients who received
nusinersen and performed daily physical therapy (study group) to those of the
control group, who received only nusinersen therapy. Results: Motor
skill improvements were statistically significantly (p