Publicaciones
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Tifft CJ, Batsu I, Giugliani R, Goyeau H, Hahn A, Jones SA, Minini P, Nakashima I, O'Callaghan-Gordo M, Perlman S, Thibault N, Kowthalam MU, Zheng R and Cox TM.
Venglustat in GM2 Gangliosidoses and Related Disorders: Results of the AMETHIST Randomized Controlled and Basket Trials.
GENETICS IN MEDICINE . : 101615-101615.
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Gavazzi F, Patel V, Erler JA, Charsar B, Vaia Y, Sevagamoorthy A, Vincent A, Woidill S, Wassmer E, Houlden H, Garcia-Cazorla A, Tonduti D, Wolf NI, Van der Knaap M, Bernard G, Adang LA and Vanderver A.
Critical Functional Domains in Pediatric Onset TUBB4A-Related Leukodystrophy: A Clinical and Caregiver's Perspective.
PEDIATRIC NEUROLOGY . 173: 156-165.
