Update zur medikamentösen Behandlung der spinalen Muskelatrophie
Author:
Publisher
Springer Science and Business Media LLC
Subject
Psychiatry and Mental health,Neurology (clinical),Neurology,General Medicine
Link
https://link.springer.com/content/pdf/10.1007/s00115-023-01462-y.pdf
Reference29 articles.
1. Wirth B (2021) Spinal muscular atrophy: in the challenge lies a solution. Trends Neurosci 44(4):306–322
2. Groen EJN, Talbot K, Gillingwater TH (2018) Advances in therapy for spinal muscular atrophy: promises and challenges. Nat Rev Neurol. https://doi.org/10.1038/nrneurol.2018.4
3. Vill K, Schwartz O, Blaschek A, Gläser D, Nennstiel U, Wirth B, Burggraf S, Röschinger W, Becker M, Czibere L et al (2021) Newborn screening for spinal muscular atrophy in Germany: clinical results after 2 years. Orphanet J Rare Dis 16(1):153
4. Müller-Felber W, Blaschek A, Schwartz O, Gläser D, Nennstiel U, Brockow I, Wirth B, Burggraf S, Röschinger W, Becker M et al (2023) Newbornscreening SMA - from pilot project to nationwide screening in Germany. J Neuromuscul Dis 10(1):55–65
5. Ziegler A, Wilichowski E, Schara U, Hahn A, Müller-Felber W, Johannsen J, von Moers A, von der von Hagen M, Stoltenburg C, Saffari A et al (2020) Recommendations for gene therapy of spinal muscular atrophy with onasemnogene abeparvovec-AVXS-101 : Consensus paper of the German representatives of the Society for Pediatric Neurology (GNP) and the German treatment centers with collaboration of the medical scientific advisory board of the German Society for Muscular Diseases (DGM). Nervenarzt 91(6):518–529
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