A double-blind, placebo-controlled, randomized trial of PXT3003 for the treatment of Charcot–Marie–Tooth type 1A

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Authors

Shahram Attarian · Hôpital de la Timone

Peter Young

Thomas H. Brannagan · Columbia University Irving Medical Center

David Adams · Bicêtre Hospital

Philip Van Damme · VIB-KU Leuven Center for Brain & Disease Research

Florian P. Thomas · Hackensack University Medical Center

Carlos Casanovas · Bellvitge University Hospital

Abstract

The high-dose group demonstrated a statistically significant improvement in the primary endpoint and a good safety profile. Overall, high-dose PXT3003 is a promising treatment option for patients with Charcot-Marie-Tooth disease type 1A.

Topics & Keywords

Hereditary Neurological Disorders Peripheral Neuropathies and Disorders Neurological diseases and metabolism

UN Sustainable Development Goals

Good health and well-being

Publication Details

Published in: Orphanet Journal of Rare Diseases

Volume 16, Issue 1, pp. 433-433

DOI: 10.1186/s13023-021-02040-8

Field-Weighted Citation Impact: 4.02