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...

Πλήρης περιγραφή

Αποθηκεύτηκε σε:
Λεπτομέρειες βιβλιογραφικής εγγραφής
Τόπος έκδοσης:Orphanet J Rare Dis
Κύριοι συγγραφείς: 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
Μορφή: Artigo
Γλώσσα:Inglês
Έκδοση: BioMed Central 2014
Θέματα:
Research
Διαθέσιμο Online:https://ncbi.nlm.nih.gov/pmc/articles/PMC4311411/
https://ncbi.nlm.nih.gov/pubmed/25519680
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1186/s13023-014-0199-0
Ετικέτες: Προσθήκη ετικέτας
Δεν υπάρχουν, Καταχωρήστε ετικέτα πρώτοι!

Παρόμοια τεκμήρια