Population-based analysis of survival in spinal muscular atrophy.
Camiel A WijngaardeMarloes StamLouise A M OttoRuben P A van EijkInge CuppenEsther S VeldhoenLeonard H van den BergRenske I WadmanLudo W van der PolPublished in: Neurology (2020)
Shortened endpoint-free survival is an important characteristic of SMA types 1 and 2a, but not types 2b, 3, and 4. For SMA types 1c and 2a, the age at which initiation of mechanical ventilation is necessary may be a more suitable endpoint than the arbitrarily set 16 h/d.