Dang, U.J., Damsker, J.M., …, Monduy, M., et al (2024). Efficacy and Safety of Vamorolone Over 48 Weeks in Boys with Duchenne Muscular Dystrophy: A Randomized Controlled Trial.
Neurology,
102(5), e208112.
View in Pubmed
Chiriboga, C.A., Bruno, C., Duong, T., et al (2023). Risdiplam in Patients Previously Treated with Other Therapies for Spinal Muscular Atrophy: An Interim Analysis from the JEWELFISH Study.
Neurology and therapy,
12 (2), 543-557.
View in Pubmed
Guglieri, M., Clemens, P.R., …, Monduy, M., et al (2022). Efficacy and Safety of Vamorolone vs Placebo and Prednisone Among Boys With Duchenne Muscular Dystrophy: A Randomized Clinical Trial.
JAMA neurology,
79(10), 1005-1014.
View in Pubmed
Klopstock, T., Videnovic, A. ..., Monduy, M., et al (2021). Fosmetpantotenate Randomized Controlled Trial in Pantothenate Kinase-Associated Neurodegeneration.
Movement disorders,
36(6), 1342-1352.
View in Pubmed