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

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 Schwann cell differentiation and dysmyelination, eventually leading to axonal s...

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Detalles Bibliográficos
Autores principales: 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
Formato: Online Artículo Texto
Lenguaje:English
Publicado: BioMed Central 2014
Materias:
Research
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4311411/
https://www.ncbi.nlm.nih.gov/pubmed/25519680
http://dx.doi.org/10.1186/s13023-014-0199-0

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