Randomized trial of thymectomy in myasthenia gravis. New England Journal of Medicine, v. 375, n. 6, p. 511-522, 2016.
A Review of Tafamidis for the Treatment of Transthyretin-Related Amyloidosis
Diagnosis, Prognosis, and Therapy of Transthyretin Amyloidosis Morie A. Gertz, MD,* Merrill D. Benson, MD,y Peter J. Dyck, MD,z Martha Grogan, MD,x Terresa Coelho, MD,k Marcia Cruz, MD,{ John L. Berk, MD,# Journal of the American College of Cardiology, vol 66, número 21, 2015. 2451-66. ISSN 0735-1097
Guideline of transthyretin-related hereditary amyloidosis for clinicians. Ando y, coelho t, berk jl, cruz mw, ericzon bg, ikeda s, lewis wd, obici l, planté-bordeneuve v, rapezzi c, said g, salvi f. Orphanet J Rare Dis. 2013 Feb 20;8:31. doi: 10.1186/1750-1172-8-31.
Motor Neuron Disease and Acquired Axonal Neuropathy Association in HIV Infection+
Regional differences and similarities of familial amyloidotic polyneuropathy (FAP) presentation in Brazil. Márcia Waddington Cruz Amyloid, 2012; 19(S1): 65–67
Early intervention with tafamidis provides long-term benefit in delaying neurological progression in patients with transthyretin familial amyloid polyneuropathy.
Lond-term effects of tafamidis for the treatment of transthyretirn familial amyloid polyneuropathy Teresa Coelho, Luis F maia, Ana Martins da Silva, Márcia W Cruz, Violaine Planté-Bordeneuve, Ole B Suhr, Isabel Conceição, Hartmut H J Schimidt, Pedro Trigo, Jeffery W Kelly, Richard Labaudinière, Jason Chan, Jell Packman, Donna R Grogan. Journal of Neurology 10.1007 s00415-013-7051-7, 22 AUGUST 2013.
Structure-based analysis of A19D, a variant of transthyretin involved in familial amyloid cardiomyopathy.Ferreira P, Sant’Anna R, Varejão N, Lima C, Novis S, Barbosa RV, Caldeira CM, Rumjanek FD, Ventura S, Cruz MW, Foguel D. PLoS One. 2013 Dec 17;8(12):e82484. doi: 10.1371/journal.pone.0082484. eCollection 2013. Erratum in: PLoS One. 2013;8(12).
Tafamidis for transthyretin familial amyloid polyneuropathy A randomized, controlled trial Teresa Coelho, MD, Luis F. Maia, MD, Ana Martins da Silva, MD, Marcia Waddington Cruz, MD, Violaine Planté-Bordeneuve, MD, Pierre Lozeron, MD, Ole B. Suhr, MD, Josep M. Campistol, MD, Isabel Maria Conceição, MD, Hartmut H.-J. Schmidt, MD, Pedro Trigo, MD, Jeffery W. Kelly, PhD, Richard Labaudinière, PhD, Jason Chan, PhD, Jeff Packman, MBA, Amy Wilson, PhD and Donna R. Grogan, MD. Neurology, 2012; 79:785-792.
The phenotypical expression of an European inherithed TTR amyloidosis in Brazil. Márcia Waddington Cruz, Débora Foguel, Amanda Berensztejn, Roberto Pedrosa, Priscila Ferreira da Silva.. Orphanet Journal of Rare Diseases 2015, 10(Suppl1):O7.
Genotype and Phenotype of Transthyretin Cardiac Amyloidosis: THAOS (Transthyretin Amyloid Outcome Survey) Mathew S. Maurer, MD,a Mazen Hanna, MD,b Martha Grogan, MD,c Angela Dispenzieri, MD,c Ronald Witteles, MD,d Brian Drachman, MD,e Daniel P. Judge, MD,f Daniel J. Lenihan, MD,g Stephen S. Gottlieb, MD,h Sanjiv J. Shah, MD,i D. Eric Steidley, MD,j Hector Ventura, MD,k Srinivas Murali, MD,l Marc A. Silver, MD,m Daniel Jacoby, MD,n Savitri Fedson, MD,o Scott L. Hummel, MD,p,q Arnt V. Kristen, MD,r Thibaud Damy, MD, PHD,s Violaine Planté-Bordeneuve, MD, PHD,s Teresa Coelho, MD,t Rajiv Mundayat, MS,u Ole B. Suhr, MD,v Márcia Waddington Cruz, MD,w Claudio Rapezzi, MD,x on behalf of the THAOS Investigators JOURNAL OF THE AMERICAN COLLEGE OF CARDIOLOGY VOL. 68, NO. 2, 2016 ª 2016 BY THE AMERICAN COLLEGE OF CARDIOLOGY FOUNDATION ISSN
Liver transplantation for familial amyloidotic polyneuropathy.M. cruz, m. saporta, c. andré, s. novis, j. ribeiro. Muscle & Nerve, Vol 36, Number 4, October 2007, Pg 571.
Cruz, M., et al. "Electroneuromyography study before and after liver transplantation in Brazilian familial amyloidotic polyneuropathy type 1 patients." JOURNAL OF NEUROLOGY. Vol. 251. PO BOX 10 04 62, D-64204 DARMSTADT, GERMANY: DR DIETRICH STEINKOPFF VERLAG, 2004.
Cruz, M., et al. "Clinical survey before and after liver transplantation in Brazilian familial amyloidotic polyneuropathy type 1 patients." JOURNAL OF NEUROLOGY. Vol. 251. PO BOX 10 04 62, D-64204 DARMSTADT, GERMANY: DR DIETRICH STEINKOPFF VERLAG, 2004.
Cruz, M. W., et al. "Ten-year experience with liver transplantation for familial amyloidotic polyneuropathy type I in Rio de Janeiro, Brazil." JOURNAL OF NEUROLOGY. Vol. 254. PO BOX 10 04 62, D-64204 DARMSTADT, GERMANY: DR DIETRICH STEINKOPFF VERLAG, 2007.
A landmark clinical trial of a novel small molecule transthyretin (TTR) stabiliser, Fx-1006 A, in patients with TTR amyloid polyneuropathy: a phase II/III, randomised, doulble blind, placebo-controled study. M. waddington cruz, t. coelho, l. maia, a. martins da silva, v. planté-bordeneuve, o. suhr, j. campistol, i. conceição, h. schimidt, p. trigo, d. cros, d. roy grogan, j. packman. P. 432. Journal of Neurology, Volume 255. Supplement 2. June 2008. pg 107.
Correlation of. clinical outcomes and disease burden in patients with transthyretin (TTR) amyloid polyneuropathy: sutdy Fx-005, a landmark clinical trial of Fx-1006 A, a novel samll molecule TTR stabiliser. T. coelho, m. waddington cruz,, v. planté-bordeneuve, d. cros, d. roy grogan, j. packman. P. 318. Journal of Neurology, Volume 255. Supplement 2. June 2008. pg 78.
On the origin of the transthyretin Val30Met familial amyloid polyneuropathy. Zaros c, genin e, hellman u, saporta ma, languille l, cruz mw, shur o, misrahi m, plante-bordeneuve v. Ann Hum Genet 2008; 72:478-484.
ATTR transplantation consensus pannel 2009. SR Zeldenrust, M Skinner, M Benson, Y Ando, A Stangou, M Waddington Cruz, T Coelho, L Obici, BG Ericzon, D Samuel, AD Pinna, SI Ikeda, OB Suhr. Amyloid. The Journal of Protein Folding Disorders, volume 17, Supplement 1, April 2010. ISSN 1350-6129. Pgs 74-75
An evaluation of the quality of life in symptomatic patients in the Transthyretin Amyloidosis Outcomes Survey (THAOS). Journal of Neurology. 258():S25, May 2011. I. Conceição; T. Coelho; V. Planté-Bordeneuve; M. Waddington Cruz; B. Ericzon; R. Falk; S. Ikeda; M. Mauer; O. Suhr; Y. Ando; A. Mazzeo; D. Grogan.
Cruz, Márcia Waddington, et al. "Demographics and Neurologic Function in Early-and Late-onset Symptomatic Patients in the Transthyretin Amyloidosis Outcomes Survey (THAOS)." Journal of the Peripheral Nervous System 16 (2011): S27.
Early-treatment Effects Of Tafamidis In Transthyretin Type Familial Amyloid Polyneuropathy. Journal of the Peripheral Nervous System, v. 16, p. S24-S25, 2011.
Interim Results from Phase II Trial on ALN-TTR02, a novel RNAi Therapeutic for the treatment of Familial Amyloidotic Polyneuropathy. T,Suhr O, Conceição I, Waddington Cruz M, Schimidt H, Campistol J, Pouget J, Buades J, Falzone R, Harrop J, De Frutos R, Butler J, Cehelsky J, Nochur S, Vaishanaw A, Gollob J. Volume 18, Supplement 2, June 2013.
Evaluation Of Modifications Of Nis+ 7 Score In Oligonucleotide Trials In Ttr Fap. Journal of the Peripheral Nervous System, v. 20, n. 2, p. 132-133, 2015.
Analysis of Transthyretinfamilial amyloid polyneuropathy disease progression in symptomatic V30Mpatients treated with tafamidis or liver tarnsplantation compared to untreatedpatients: an analysis from the transthyretin amyloidosis outcomes survey(THAOS) registry.
Identification of a new variant of TTRinvolved in familial amyloid cardiomyopathy (FAC) in Brazil:from the patient tothe protein.
Phase2 open-label extention (OLE) study of patisiran, an investigational siRNA agentfor familial amyloidotic polyneuropathy (FAP)
Difference of penetrance in transthyretin amyloid neuropathies across families from European and Barzilian origin. O 147. V. Planté – Bordeneuve, U. Hellman, M. Saporta, A. Ferrera., C. Zaros, O. Suhr. M. Waddington Cruz., M. Mirashi. G. Said, Bonaiti, Pelllié. .Journal of Neurology, Volume 253. Supplment 2. May 2006.
Zaros C, Genin E, Hellman U, Saporta MA, Languille L, Cruz MW, Shur O, Misrahi M, Plante-Bordeneuve V. On the origin of the transthyretin Val30Met familial amyloid polyneuropathy. Ann Hum Genet 2008; 72:478-484.
Penetrance estimation of TTR familial amyloid polyneuropathy (type I) in Brazilian families. M.A.C. Saporta; C. Zaros, M. W. Cruz, C. André, M. Misrahi, C. Bonati- Pellié and V. Planté-Bordeneuve. European Journal of Neurology, 2009.16: 337-341.
Discordant expression of familial amuloid polyneuropathy in homozygotic Brazilian twins. Mário andré da cunha saporta, v. planté-bordeneuve, m. misrahi, m. w. cruz. Amyloid, 2009; 16(1): 38-41
Tafamidis (FX-1006 A): a first-in-class disease- modifying therapy for transthyretin familial amyloid. T. Coelho, L. Maia, A. Martins da Silva, M. Waddington Cruz, V. Planté-Bordeneuve, P Lozeron, OB Suhr, J Campistol. IM Conceição, H Schmidt. P Trigo, J Packman, DR Grogan, pg 75-76. Amyloid. The Journal of Protein Folding Disorders, volume 17, Supplement 1, April 2010. ISSN 1350-6129.
Yukio Ando Y, Teresa Coelho T, Berk JL, Waddington Cruz M, Ericzon B-G, Ikeda S, Lewis WD, Obici L, Planté-Bordeneuve V, Rapezzi C, Said G, Salvi F. Guideline of transthyretin-related hereditary amyloidosis for clinicians. Orphanet Journal of Rare Diseases 2013, 8:31 (doi:10.1186/1750-1172-8-31)
A Review of Tafamidis for the Treatment of Transthyretin-Related Amyloidosis Márcia Waddington Cruz1 and Merril D. Benson2 Neurology and Therapy 2015:31 DOI: 10.1007/s40120-015-0031-3 Published online: 15 August 2015.
Efficacy and safety of patisiran for familial amyloidotic polyneuropathy: a phase II multi-dose study Ole B Suhr1*, Teresa Coelho2 , Juan Buades3 , Jean Pouget4 , Isabel Conceicao5 , John Berk6 , Hartmut Schmidt7 , Márcia Waddington-Cruz8 , Josep M. Campistol9 , Brian R. Bettencourt10, Akshay Vaishnaw10, Jared Gollob10 and David Adams. Suhr et al. Orphanet Journal of Rare Diseases (2015) 10:109 DOI 10.1186/s13023-015-0326-6, pages 1-9.
Penetrance estimation and haplotype analysis of TTR val30met mutation on Brazilian families with familial amyloidotic polyneuropathy type I. M. saporta, c. andré, c. zaros, m. misrahi, g. said, e. génin, c bonaiti-pellié, v. planté-bordeneuve, mw cruz. Journal of Neurology, Volume 254. Supplement 3. May 2007, O 81, pg 18.
Journal of the American College of Cardiology Vol. 68, No. 2, 2016 ª 2016 By the American College of Cardiology Foundation ISSN 0735-1097/$36.00 Published by Sevier
