Systematic Review of Newborn Screening Programmes for Spinal Muscular Atrophy

• Main Authors: Katy Cooper (Author), Gamze Nalbant (Author), Anthea Sutton (Author), Sue Harnan (Author), Praveen Thokala (Author), Jim Chilcott (Author), Alisdair McNeill (Author), Alice Bessey (Author)
• Format: Book
• Published: MDPI AG, 2024-07-01T00:00:00Z.
• Subjects: article
• Online Access: Connect to this object online.

Spinal muscular atrophy (SMA) is a genetic neuromuscular disorder causing the degeneration of motor neurons in the spinal cord. Recent studies suggest greater effectiveness of treatment in the presymptomatic stage. This systematic review synthesises findings from 37 studies (and 3 overviews) of newb...