A double-blind, placebo-controlled, randomized trial of PXT3003 for the treatment of Charcot-Marie-Tooth type 1A.
Shahram AttarianPeter YoungThomas H BrannaganDavid AdamsPhilip Van DammeFlorian P ThomasCarlos CasanovasCéline TardMaggie C WalterYann PéréonDavid WalkAmro StinoMarianne de VisserCamiel VerhammeAnthony AmatoGregory CarterLaurent MagyJeffrey M StatlandKevin FelicePublished in: Orphanet journal of rare diseases (2021)
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.