Attarian, S., Young, P., Brannagan, T. H., Adams, D., Van Damme, P., Thomas, F. P., Casanovas, C., Kafaie, J., Tard, C., Walter, M. C., Péréon, Y., Walk, D., Stino, A., de Visser, M., Verhamme, C., Amato, A., Carter, G., Magy, L., Statland, J. M., & Felice, K. (2021). A double-blind, placebo-controlled, randomized trial of PXT3003 for the treatment of Charcot–Marie–Tooth type 1A. Orphanet Journal of Rare Diseases, 16(1). https://doi.org/10.1186/s13023-021-02040-8
Subjects:
Genetic Basis of Neuropathies and Related Disorders
(OpenAlex Topic)
Guillain-Barré Syndrome and Related Neuropathies
(OpenAlex Topic)
Neurodegeneration with Brain Iron Accumulation
(OpenAlex Topic)
Charcot-Marie-Tooth Disease
(MeSH)
Publication Type:
Article
Unique ID:
10.1186/s13023-021-02040-8
PMID:
Journal:
Publication Date:
Data Source:
Scopus