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347 results

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A double-blind, placebo-controlled, randomized trial of PXT3003 for the treatment of Charcot-Marie-Tooth type 1A.
Attarian S, Young P, Brannagan TH, Adams D, Van Damme P, Thomas FP, Casanovas C, Kafaie J, Tard C, Walter MC, Péréon Y, Walk D, Stino A, de Visser M, Verhamme C, Amato A, Carter G, Magy L, Statland JM, Felice K. Attarian S, et al. Orphanet J Rare Dis. 2021 Oct 16;16(1):433. doi: 10.1186/s13023-021-02040-8. Orphanet J Rare Dis. 2021. PMID: 34656144 Free PMC article. Clinical Trial.
Effect of ascorbic acid in patients with Charcot-Marie-Tooth disease type 1A: a multicentre, randomised, double-blind, placebo-controlled trial.
Micallef J, Attarian S, Dubourg O, Gonnaud PM, Hogrel JY, Stojkovic T, Bernard R, Jouve E, Pitel S, Vacherot F, Remec JF, Jomir L, Azabou E, Al-Moussawi M, Lefebvre MN, Attolini L, Yaici S, Tanesse D, Fontes M, Pouget J, Blin O. Micallef J, et al. Among authors: attarian s. Lancet Neurol. 2009 Dec;8(12):1103-10. doi: 10.1016/S1474-4422(09)70260-1. Epub 2009 Oct 7. Lancet Neurol. 2009. PMID: 19818690 Clinical Trial.
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. 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.
Genotype-phenotype correlation and course of transthyretin familial amyloid polyneuropathies in France.
Mariani LL, Lozeron P, Théaudin M, Mincheva Z, Signate A, Ducot B, Algalarrondo V, Denier C, Adam C, Nicolas G, Samuel D, Slama MS, Lacroix C, Misrahi M, Adams D; French Familial Amyloid Polyneuropathies Network (CORNAMYL) Study Group. Mariani LL, et al. Ann Neurol. 2015 Dec;78(6):901-16. doi: 10.1002/ana.24519. Epub 2015 Oct 7. Ann Neurol. 2015. PMID: 26369527 Free PMC article.
A randomized, placebo-controlled, double-blind phase IIb trial evaluating the safety and efficacy of tirasemtiv in patients with amyotrophic lateral sclerosis.
Shefner JM, Wolff AA, Meng L, Bian A, Lee J, Barragan D, Andrews JA; BENEFIT-ALS Study Group. Shefner JM, et al. Amyotroph Lateral Scler Frontotemporal Degener. 2016 Jul-Aug;17(5-6):426-435. doi: 10.3109/21678421.2016.1148169. Epub 2016 Mar 16. Amyotroph Lateral Scler Frontotemporal Degener. 2016. PMID: 26982815 Clinical Trial.
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. 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.
Skin Biopsy Findings in Patients With CMT1A: Baseline Data From the CLN-PXT3003-01 Study Provide New Insights Into the Pathophysiology of the Disorder.
Duchesne M, Danigo A, Richard L, Vallat JM, Attarian S, Gonnaud PM, Lacour A, Péréon Y, Stojkovic T, Nave KA, Bertrand V, Nabirotchkin S, Cohen D, Demiot C, Magy L. Duchesne M, et al. Among authors: attarian s. J Neuropathol Exp Neurol. 2018 Apr 1;77(4):274-281. doi: 10.1093/jnen/nly001. J Neuropathol Exp Neurol. 2018. PMID: 29408953
347 results