Browsing FAS Scholarly Articles by Keyword "CMT1A"

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    • An exploratory randomised double-blind and placebo-controlled phase 2 study of a combination of baclofen, naltrexone and sorbitol (PXT3003) in patients with Charcot-Marie-Tooth disease type 1A 

      Attarian, Shahram; Vallat, Jean-Michel; Magy, Laurent; Funalot, Benoît; Gonnaud, Pierre-Marie; Lacour, Arnaud; Péréon, Yann; Dubourg, Odile; Pouget, Jean; Micallef, Joëlle; Franques, Jérôme; Lefebvre, Marie-Noëlle; Ghorab, Karima; Al-Moussawi, Mahmoud; Tiffreau, Vincent; Preudhomme, Marguerite; Magot, Armelle; Leclair-Visonneau, Laurène; Stojkovic, Tanya; Bossi, Laura; Lehert, Philippe; Gilbert, Walter; Bertrand, Viviane; Mandel, Jonas; Milet, Aude; Hajj, Rodolphe; Boudiaf, Lamia; Scart-Grès, Catherine; Nabirotchkin, Serguei; Guedj, Mickael; Chumakov, Ilya; Cohen, Daniel (BioMed Central, 2014)
      Background: Charcot-Marie-Tooth type 1A disease (CMT1A) is a rare orphan inherited neuropathy caused by an autosomal dominant duplication of a gene encoding for the structural myelin protein PMP22, which induces abnormal ...