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A phase II, open-label, extension study of long-term patisiran treatment in patients with hereditary transthyretin-mediated (hATTR) amyloidosis.
Coelho T, Adams D, Conceição I, Waddington-Cruz M, Schmidt HH, Buades J, Campistol J, Berk JL, Polydefkis M, Wang JJ, Chen J, Sweetser MT, Gollob J, Suhr OB. Coelho T, et al. Among authors: berk jl. Orphanet J Rare Dis. 2020 Jul 8;15(1):179. doi: 10.1186/s13023-020-01399-4. Orphanet J Rare Dis. 2020. PMID: 32641071 Free PMC article. Clinical Trial.
The Diflunisal Trial: study accrual and drug tolerance.
Berk JL, Suhr OB, Sekijima Y, Yamashita T, Heneghan M, Zeldenrust SR, Ando Y, Ikeda S, Gorevic P, Merlini G, Kelly JW, Skinner M, Bisbee AB, Dyck PJ, Obici L; Familial Amyloidosis Consortium. Berk JL, et al. Amyloid. 2012 Jun;19 Suppl 1:37-8. doi: 10.3109/13506129.2012.678509. Epub 2012 May 2. Amyloid. 2012. PMID: 22551208 Clinical Trial.
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. Ando Y, et al. Among authors: berk jl. Orphanet J Rare Dis. 2013 Feb 20;8:31. doi: 10.1186/1750-1172-8-31. Orphanet J Rare Dis. 2013. PMID: 23425518 Free PMC article. Review.
Repurposing diflunisal for familial amyloid polyneuropathy: a randomized clinical trial.
Berk JL, Suhr OB, Obici L, Sekijima Y, Zeldenrust SR, Yamashita T, Heneghan MA, Gorevic PD, Litchy WJ, Wiesman JF, Nordh E, Corato M, Lozza A, Cortese A, Robinson-Papp J, Colton T, Rybin DV, Bisbee AB, Ando Y, Ikeda S, Seldin DC, Merlini G, Skinner M, Kelly JW, Dyck PJ; Diflunisal Trial Consortium. Berk JL, et al. JAMA. 2013 Dec 25;310(24):2658-67. doi: 10.1001/jama.2013.283815. JAMA. 2013. PMID: 24368466 Free PMC article. Clinical Trial.
Diagnosis, Prognosis, and Therapy of Transthyretin Amyloidosis.
Gertz MA, Benson MD, Dyck PJ, Grogan M, Coelho T, Cruz M, Berk JL, Plante-Bordeneuve V, Schmidt HHJ, Merlini G. Gertz MA, et al. Among authors: berk jl. J Am Coll Cardiol. 2015 Dec 1;66(21):2451-2466. doi: 10.1016/j.jacc.2015.09.075. J Am Coll Cardiol. 2015. PMID: 26610878 Free article. Review.
Assessing mNIS+7Ionis and international neurologists' proficiency in a familial amyloidotic polyneuropathy trial.
Dyck PJ, Kincaid JC, Dyck PJB, Chaudhry V, Goyal NA, Alves C, Salhi H, Wiesman JF, Labeyrie C, Robinson-Papp J, Cardoso M, Laura M, Ruzhansky K, Cortese A, Brannagan TH 3rd, Khoury J, Khella S, Waddington-Cruz M, Ferreira J, Wang AK, Pinto MV, Ayache SS, Benson MD, Berk JL, Coelho T, Polydefkis M, Gorevic P, Adams DH, Plante-Bordeneuve V, Whelan C, Merlini G, Heitner S, Drachman BM, Conceição I, Klein CJ, Gertz MA, Ackermann EJ, Hughes SG, Mauermann ML, Bergemann R, Lodermeier KA, Davies JL, Carter RE, Litchy WJ. Dyck PJ, et al. Among authors: berk jl. Muscle Nerve. 2017 Nov;56(5):901-911. doi: 10.1002/mus.25563. Epub 2017 Apr 7. Muscle Nerve. 2017. PMID: 28063170 Free PMC article. Clinical Trial.
Patisiran, an RNAi Therapeutic, for Hereditary Transthyretin Amyloidosis.
Adams D, Gonzalez-Duarte A, O'Riordan WD, Yang CC, Ueda M, Kristen AV, Tournev I, Schmidt HH, Coelho T, Berk JL, Lin KP, Vita G, Attarian S, Planté-Bordeneuve V, Mezei MM, Campistol JM, Buades J, Brannagan TH 3rd, Kim BJ, Oh J, Parman Y, Sekijima Y, Hawkins PN, Solomon SD, Polydefkis M, Dyck PJ, Gandhi PJ, Goyal S, Chen J, Strahs AL, Nochur SV, Sweetser MT, Garg PP, Vaishnaw AK, Gollob JA, Suhr OB. Adams D, et al. Among authors: berk jl. N Engl J Med. 2018 Jul 5;379(1):11-21. doi: 10.1056/NEJMoa1716153. N Engl J Med. 2018. PMID: 29972753 Free article. Clinical Trial.
Inotersen Treatment for Patients with Hereditary Transthyretin Amyloidosis.
Benson MD, Waddington-Cruz M, Berk JL, Polydefkis M, Dyck PJ, Wang AK, Planté-Bordeneuve V, Barroso FA, Merlini G, Obici L, Scheinberg M, Brannagan TH 3rd, Litchy WJ, Whelan C, Drachman BM, Adams D, Heitner SB, Conceição I, Schmidt HH, Vita G, Campistol JM, Gamez J, Gorevic PD, Gane E, Shah AM, Solomon SD, Monia BP, Hughes SG, Kwoh TJ, McEvoy BW, Jung SW, Baker BF, Ackermann EJ, Gertz MA, Coelho T. Benson MD, et al. Among authors: berk jl. N Engl J Med. 2018 Jul 5;379(1):22-31. doi: 10.1056/NEJMoa1716793. N Engl J Med. 2018. PMID: 29972757 Free article. Clinical Trial.
Tafamidis Treatment for Patients with Transthyretin Amyloid Cardiomyopathy.
Maurer MS, Schwartz JH, Gundapaneni B, Elliott PM, Merlini G, Waddington-Cruz M, Kristen AV, Grogan M, Witteles R, Damy T, Drachman BM, Shah SJ, Hanna M, Judge DP, Barsdorf AI, Huber P, Patterson TA, Riley S, Schumacher J, Stewart M, Sultan MB, Rapezzi C; ATTR-ACT Study Investigators. Maurer MS, et al. N Engl J Med. 2018 Sep 13;379(11):1007-1016. doi: 10.1056/NEJMoa1805689. Epub 2018 Aug 27. N Engl J Med. 2018. PMID: 30145929 Free article. Clinical Trial.
152 results