Systematic Review of Presymptomatic Treatment for Spinal Muscular Atrophy

• मुख्य लेखकों: Katy Cooper (लेखक), Gamze Nalbant (लेखक), Anthea Sutton (लेखक), Sue Harnan (लेखक), Praveen Thokala (लेखक), Jim Chilcott (लेखक), Alisdair McNeill (लेखक), Alice Bessey (लेखक)
• स्वरूप: पुस्तक
• प्रकाशित: MDPI AG, 2024-08-01T00:00:00Z.
• विषय: article
• ऑनलाइन पहुंच: 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...