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Newborn screening in metachromatic leukodystrophy - European consensus-based recommendations on clinical management.
Eur J Paediatr Neurol. 2024 Mar;49:141-154. doi: 10.1016/j.ejpn.2024.03.003. Epub 2024 Mar 9.
Eur J Paediatr Neurol. 2024.
PMID: 38554683
Free article.
Core protocol development for phase 2/3 clinical trials in the leukodystrophy vanishing white matter: a consensus statement by the VWM consortium and patient advocates.
Schoenmakers DH, Leferink PS, Vanderver A, Bonkowsky JL, Krägeloh-Mann I, Bernard G, Bertini E, Fatemi A, Fogel BL, Wolf NI, Skwirut D, Buck A, Holberg B, Saunier-Vivar EF, Rauner R, Dekker H, van Bokhoven P, Stellingwerff MD, Berkhof J, van der Knaap MS.
Schoenmakers DH, et al. Among authors: saunier vivar ef.
BMC Neurol. 2023 Aug 17;23(1):305. doi: 10.1186/s12883-023-03354-9.
BMC Neurol. 2023.
PMID: 37592248
Free PMC article.
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Therapy Trial Design in Vanishing White Matter: An Expert Consortium Opinion.
van der Knaap MS, Bonkowsky JL, Vanderver A, Schiffmann R, Krägeloh-Mann I, Bertini E, Bernard G, Fatemi SA, Wolf NI, Saunier-Vivar E, Rauner R, Dekker H, van Bokhoven P, van de Ven P, Leferink PS.
van der Knaap MS, et al. Among authors: saunier vivar e.
Neurol Genet. 2022 Feb 2;8(2):e657. doi: 10.1212/NXG.0000000000000657. eCollection 2022 Apr.
Neurol Genet. 2022.
PMID: 35128050
Free PMC article.
Review.
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