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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. Among authors: walter mc. 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.
Selected items from the Charcot-Marie-Tooth (CMT) Neuropathy Score and secondary clinical outcome measures serve as sensitive clinical markers of disease severity in CMT1A patients.
Mannil M, Solari A, Leha A, Pelayo-Negro AL, Berciano J, Schlotter-Weigel B, Walter MC, Rautenstrauss B, Schnizer TJ, Schenone A, Seeman P, Kadian C, Schreiber O, Angarita NG, Fabrizi GM, Gemignani F, Padua L, Santoro L, Quattrone A, Vita G, Calabrese D; CMT-TRIAAL/CMT-TRAUK Group; Young P, Laurà M, Haberlová J, Mazanec R, Paulus W, Beissbarth T, Shy ME, Reilly MM, Pareyson D, Sereda MW. Mannil M, et al. Among authors: walter mc. Neuromuscul Disord. 2014 Nov;24(11):1003-17. doi: 10.1016/j.nmd.2014.06.431. Epub 2014 Jun 19. Neuromuscul Disord. 2014. PMID: 25085517 Clinical Trial.
Biomarkers predict outcome in Charcot-Marie-Tooth disease 1A.
Fledrich R, Mannil M, Leha A, Ehbrecht C, Solari A, Pelayo-Negro AL, Berciano J, Schlotter-Weigel B, Schnizer TJ, Prukop T, Garcia-Angarita N, Czesnik D, Haberlová J, Mazanec R, Paulus W, Beissbarth T, Walter MC, Triaal C, Hogrel JY, Dubourg O, Schenone A, Baets J, De Jonghe P, Shy ME, Horvath R, Pareyson D, Seeman P, Young P, Sereda MW. Fledrich R, et al. Among authors: walter mc. J Neurol Neurosurg Psychiatry. 2017 Nov;88(11):941-952. doi: 10.1136/jnnp-2017-315721. Epub 2017 Aug 31. J Neurol Neurosurg Psychiatry. 2017. PMID: 28860329 Free PMC article.
Cost of illness in Charcot-Marie-Tooth neuropathy: Results from Germany.
Schorling E, Thiele S, Gumbert L, Krause S, Klug C, Schreiber-Katz O, Reilich P, Nagels K, Walter MC. Schorling E, et al. Among authors: walter mc. Neurology. 2019 Apr 23;92(17):e2027-e2037. doi: 10.1212/WNL.0000000000007376. Epub 2019 Mar 27. Neurology. 2019. PMID: 30918088
226 results