Postnatal management of preterm infants with spinal muscular atrophy: experience from German newborn screening

Postnatal management of preterm infants with spinal muscular atrophy: experience from German newborn screening

Abstract Background The introduction of newborn screening (NBS) for spinal muscular atrophy (SMA) has increased the early diagnosis of 5q-associated SMA in presymptomatic and symptomatic preterm infants. National and international recommendations for treating preterms and newborns < 38 weeks of g...

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Bibliographic Details
Main Authors: Regina Trollmann, Jessika Johannsen, Katharina Vill, Cornelia Köhler, Andreas Hahn, Sabine Illsinger, Astrid Pechmann, Maja von der Hagen, Wolfgang Müller-Felber
Format: Article
Language:English
Published: BMC 2024-09-01
Series:Orphanet Journal of Rare Diseases
Subjects:
5q-SMA
Preterm infants
Spinal muscular atrophy
Newborn screening
SMA treatment
Onasemnogene Abeparvovec
Online Access:https://doi.org/10.1186/s13023-024-03362-z

Internet

https://doi.org/10.1186/s13023-024-03362-z

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