Expanding the Availability of Onasemnogene Abeparvovec to Older Patients: The Evolving Treatment Landscape for Spinal Muscular Atrophy

• Main Authors: Charlotte A. René (Author), Robin J. Parks (Author)
• Formato: Libro
• Publicado: MDPI AG, 2023-06-01T00:00:00Z.
• Subjects: article
• Acceso en liña: Connect to this object online.

Spinal muscular atrophy (SMA) is a devastating neuromuscular disorder caused by mutations in the survival of motor neuron 1 (<i>SMN1</i>) gene, which leads to a reduced level in the SMN protein within cells. Patients with SMA suffer from a loss of alpha motor neurons in the spinal cord l...