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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 S, Vallat JM, Magy L, Funalot B, Gonnaud PM, Lacour A, Péréon Y, Dubourg O, Pouget J, Micallef J, Franques J, Lefebvre MN, Ghorab K, Al-Moussawi M, Tiffreau V, Preudhomme M, Magot A, Leclair-Visonneau L, Stojkovic T, Bossi L, Lehert P, Gilbert W, Bertrand V, Mandel J, Milet A, Hajj R, Boudiaf L, Scart-Grès C, Nabirotchkin S, Guedj M, Chumakov I, Cohen D. Attarian S, et al. Among authors: hajj r. Orphanet J Rare Dis. 2014 Dec 18;9:199. doi: 10.1186/s13023-014-0199-0. Orphanet J Rare Dis. 2014. PMID: 25519680 Free PMC article. Clinical Trial.
Polytherapy with a combination of three repurposed drugs (PXT3003) down-regulates Pmp22 over-expression and improves myelination, axonal and functional parameters in models of CMT1A neuropathy.
Chumakov I, Milet A, Cholet N, Primas G, Boucard A, Pereira Y, Graudens E, Mandel J, Laffaire J, Foucquier J, Glibert F, Bertrand V, Nave KA, Sereda MW, Vial E, Guedj M, Hajj R, Nabirotchkin S, Cohen D. Chumakov I, et al. Among authors: hajj r. Orphanet J Rare Dis. 2014 Dec 10;9:201. doi: 10.1186/s13023-014-0201-x. Orphanet J Rare Dis. 2014. PMID: 25491744 Free PMC article.
Erratum to: 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 S, Vallat JM, Magy L, Funalot B, Gonnaud PM, Lacour A, Péréon Y, Dubourg O, Pouget J, Micallef J, Franques J, Lefebvre MN, Ghorab K, Al-Moussawi M, Tiffreau V, Preudhomme M, Magot A, Leclair-Visonneau L, Stojkovic T, Bossi L, Lehert P, Gilbert W, Bertrand V, Mandel J, Milet A, Hajj R, Boudiaf L, Scart-Grès C, Nabirotchkin S, Guedj M, Chumakov I, Cohen D. Attarian S, et al. Among authors: hajj r. Orphanet J Rare Dis. 2016 Jul 7;11(1):92. doi: 10.1186/s13023-016-0463-6. Orphanet J Rare Dis. 2016. PMID: 27387831 Free PMC article. No abstract available.
Synergistic PXT3003 therapy uncouples neuromuscular function from dysmyelination in male Charcot-Marie-Tooth disease type 1A (CMT1A) rats.
Prukop T, Wernick S, Boussicault L, Ewers D, Jäger K, Adam J, Winter L, Quintes S, Linhoff L, Barrantes-Freer A, Bartl M, Czesnik D, Zschüntzsch J, Schmidt J, Primas G, Laffaire J, Rinaudo P, Brureau A, Nabirotchkin S, Schwab MH, Nave KA, Hajj R, Cohen D, Sereda MW. Prukop T, et al. Among authors: hajj r. J Neurosci Res. 2020 Oct;98(10):1933-1952. doi: 10.1002/jnr.24679. Epub 2020 Jun 26. J Neurosci Res. 2020. PMID: 32588471
Combining two repurposed drugs as a promising approach for Alzheimer's disease therapy.
Chumakov I, Nabirotchkin S, Cholet N, Milet A, Boucard A, Toulorge D, Pereira Y, Graudens E, Traoré S, Foucquier J, Guedj M, Vial E, Callizot N, Steinschneider R, Maurice T, Bertrand V, Scart-Grès C, Hajj R, Cohen D. Chumakov I, et al. Among authors: hajj r. Sci Rep. 2015 Jan 8;5:7608. doi: 10.1038/srep07608. Sci Rep. 2015. PMID: 25566747 Free PMC article.
Next-generation drug repurposing using human genetics and network biology.
Nabirotchkin S, Peluffo AE, Rinaudo P, Yu J, Hajj R, Cohen D. Nabirotchkin S, et al. Among authors: hajj r. Curr Opin Pharmacol. 2020 Apr;51:78-92. doi: 10.1016/j.coph.2019.12.004. Epub 2020 Jan 22. Curr Opin Pharmacol. 2020. PMID: 31982325 Free article. Review.
71 results