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

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

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 suffering the...

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Autori principali: Shahram Attarian, Jean‐Michel Vallat, Laurent Magy, Benoît Funalot, Pierre‐Marie Gonnaud, Arnaud Lacour, Yann Péréon, O. Dubourg, Jean Pouget, Joëlle Micallef, J. Franques, Marie‐Noëlle Lefebvre, Karima Ghorab, Mahmoud Al-Moussawi, V. Tiffreau, Marguerite Preudhomme, Armelle Magot, Laurène Leclair‐Visonneau, Tanya Stojkovic, Laura Bossi, Philippe Lehert, Walter Gilbert, Viviane Bertrand, Jonas Mandel, Aude Milet, Rodolphe Hajj, Lamia Boudiaf, Catherine Scart‐Grès, Serguei Nabirotchkin, Mickaël Guedj, Ilya Chumakov, Daniel Cohen
Natura: Artigo
Lingua:inglese
Pubblicazione: 2014
Accesso online:https://doi.org/10.1186/s13023-014-0199-0
https://ojrd.biomedcentral.com/counter/pdf/10.1186/s13023-014-0199-0
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https://doi.org/10.1186/s13023-014-0199-0
https://ojrd.biomedcentral.com/counter/pdf/10.1186/s13023-014-0199-0

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