A double-blind, placebo-controlled, randomized trial of PXT3003 for the treatment of Charcot-Marie-Tooth type 1A.
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Orphanet J Rare Dis. 2021 Oct 16;16(1):433. doi: 10.1186/s13023-021-02040-8.
Orphanet J Rare Dis. 2021.
PMID: 34656144
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Clinical Trial.