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.

Autor: Schoenmakers, Daphne H.^1,2,3 (AUTHOR), Leferink, Prisca S.⁴ (AUTHOR), Vanderver, Adeline^5,6 (AUTHOR), Bonkowsky, Joshua L.^7,8 (AUTHOR), Krägeloh-Mann, Ingeborg⁹ (AUTHOR), Bernard, Geneviève^10,11 (AUTHOR), Bertini, Enrico¹² (AUTHOR), Fatemi, Ali¹³ (AUTHOR), Fogel, Brent L.¹⁴ (AUTHOR), Wolf, Nicole I.^1,2 (AUTHOR), Skwirut, Donna^15,16 (AUTHOR), Buck, Allyson¹⁶ (AUTHOR), Holberg, Brett¹⁶ (AUTHOR), Saunier-Vivar, Elise F.¹⁷ (AUTHOR), Rauner, Robert¹⁵ (AUTHOR), Dekker, Hanka¹⁸ (AUTHOR), van Bokhoven, Pieter⁴ (AUTHOR), Stellingwerff, Menno D.^1,2 (AUTHOR), Berkhof, Johannes¹⁹ (AUTHOR), van der Knaap, Marjo S.^1,2,20 (AUTHOR) ms.vanderknaap@amsterdamumc.nl

Publikováno v: BMC Neurology. 8/17/2023, Vol. 23 Issue 1, p1-15. 15p.

Patterning factors during neural progenitor induction determine regional identity and differentiation potential in vitro

Autor: Nadadhur, Aishwarya G., Leferink, Prisca S., Holmes, Dwayne, Hinz, Lisa, Cornelissen-Steijger, Paulien, Gasparotto, Lisa, Heine, Vivi M.

Publikováno v: In Stem Cell Research October 2018 32:25-34

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Cell Replacement Therapy Improves Pathological Hallmarks in a Mouse Model of Leukodystrophy Vanishing White Matter

Autor: Dooves, Stephanie, Leferink, Prisca S., Krabbenborg, Sander, Breeuwsma, Nicole, Bots, Saskia, Hillen, Anne E.J., Jacobs, Gerbren, van der Knaap, Marjo S., Heine, Vivi M.

Publikováno v: In Stem Cell Reports 5 March 2019 12(3):441-450

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