Changes in Ventilatory Support Requirements of Spinal Muscular Atrophy (SMA) Patients Post Gene-Based Therapies

• Main Authors: Panagiota Panagiotou (Author), Christina Kanaka-Gantenbein (Author), Athanasios G. Kaditis (Author)
• Formato: Libro
• Publicado: MDPI AG, 2022-08-01T00:00:00Z.
• Subjects: article
• Acceso en liña: Connect to this object online.

Spinal muscular atrophy (SMA) is a genetic neuromuscular disease resulting in global muscular weakness and, frequently, in respiratory failure and premature death. Gene-based therapies like Nusinersen are now available for patients with SMA. The aim of this review was to assess in "real world&q...