Natural history of respiratory muscle strength in spinal muscular atrophy: a prospective national cohort study.

Esther S VeldhoenCamiel A WijngaardeErik H J HulzebosRoelie M Wösten-van AsperenRenske I WadmanRuben P A van EijkFay Lynn AsselmanMarloes StamLouise A M OttoInge CuppenFeline E V ScheijmansLaura P Verweij-van den OudenrijnBart BartelsMichael A GaytantCornelis K van der EntW Ludo van der Pol

Published in: Orphanet journal of rare diseases (2022)

There are clear differences in respiratory muscle strength and its progressive decline between SMA types. We observed lower outcomes in more severe SMA types. Particularly PEF may be a suitable outcome measure for the follow-up of respiratory strength in patients with SMA. PEF declines in a rather linear pattern in all SMA types, with clear differences at baseline. These natural history data may serve as a reference for longer-term treatment efficacy assessments.

Keyphrases

respiratory tract
preterm infants
early onset
electronic health record
quality improvement
big data
machine learning
metabolic syndrome
skeletal muscle
deep learning
preterm birth
artificial intelligence
neural network