A phase 3 trial highlights betibeglogene autotemcel as a potentially curative gene therapy for severe transfusion-dependent β ...

The top 10 most-read stories published by SMA News Today in 2024 focused on symptoms, treatments, and diagnosis.

More than three years after Novartis’ plan for a speedy approval for intrathecal Zolgensma was thwarted by an FDA requirement ...

Novartis’ Phase III study has demonstrated significant improvement in motor function in children and young adults with spinal ...