Systematic Review of Presymptomatic Treatment for Spinal Muscular Atrophy

• Autori principali: Katy Cooper (Autore), Gamze Nalbant (Autore), Anthea Sutton (Autore), Sue Harnan (Autore), Praveen Thokala (Autore), Jim Chilcott (Autore), Alisdair McNeill (Autore), Alice Bessey (Autore)
• Natura: Libro
• Pubblicazione: MDPI AG, 2024-08-01T00:00:00Z.
• Soggetti: article
• Accesso online: Connect to this object online.

Spinal muscular atrophy (SMA) causes the degeneration of motor neurons in the spinal cord. Treatments including nusinersen, risdiplam, and onasemnogene abeparvovec have been shown to be effective in reducing symptoms, with recent studies suggesting greater effectiveness when treatment is initiated i...