Systematic Review of Newborn Screening Programmes for Spinal Muscular Atrophy

• Hoofdauteurs: Katy Cooper (Auteur), Gamze Nalbant (Auteur), Anthea Sutton (Auteur), Sue Harnan (Auteur), Praveen Thokala (Auteur), Jim Chilcott (Auteur), Alisdair McNeill (Auteur), Alice Bessey (Auteur)
• Formaat: Boek
• Gepubliceerd in: MDPI AG, 2024-07-01T00:00:00Z.
• Onderwerpen: article
• Online toegang: 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...