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Long-term safety and efficacy of patisiran for hereditary transthyretin-mediated amyloidosis with polyneuropathy: 12-month results of an open-label extension study.
Adams D, Polydefkis M, González-Duarte A, Wixner J, Kristen AV, Schmidt HH, Berk JL, Losada López IA, Dispenzieri A, Quan D, Conceição IM, Slama MS, Gillmore JD, Kyriakides T, Ajroud-Driss S, Waddington-Cruz M, Mezei MM, Planté-Bordeneuve V, Attarian S, Mauricio E, Brannagan TH 3rd, Ueda M, Aldinc E, Wang JJ, White MT, Vest J, Berber E, Sweetser MT, Coelho T; patisiran Global OLE study group. Adams D, et al. Among authors: quan d. Lancet Neurol. 2021 Jan;20(1):49-59. doi: 10.1016/S1474-4422(20)30368-9. Epub 2020 Nov 16. Lancet Neurol. 2021. PMID: 33212063 Clinical Trial.
Analysis of autonomic outcomes in APOLLO, a phase III trial of the RNAi therapeutic patisiran in patients with hereditary transthyretin-mediated amyloidosis.
González-Duarte A, Berk JL, Quan D, Mauermann ML, Schmidt HH, Polydefkis M, Waddington-Cruz M, Ueda M, Conceição IM, Kristen AV, Coelho T, Cauquil CA, Tard C, Merkel M, Aldinc E, Chen J, Sweetser MT, Wang JJ, Adams D. González-Duarte A, et al. Among authors: quan d. J Neurol. 2020 Mar;267(3):703-712. doi: 10.1007/s00415-019-09602-8. Epub 2019 Nov 14. J Neurol. 2020. PMID: 31728713 Free PMC article. Clinical Trial.
Correction to: Analysis of autonomic outcomes in APOLLO, a phase III trial of the RNAi therapeutic patisiran in patients with hereditary transthyretin-mediated amyloidosis.
González-Duarte A, Berk JL, Quan D, Mauermann ML, Schmidt HH, Polydefkis M, Waddington-Cruz M, Ueda M, Conceição IM, Kristen AV, Coelho T, Cauquil CA, Tard C, Merkel M, Aldinc E, Chen J, Sweetser MT, Wang JJ, Adams D. González-Duarte A, et al. Among authors: quan d. J Neurol. 2020 Mar;267(3):713-714. doi: 10.1007/s00415-020-09715-5. J Neurol. 2020. PMID: 32030522 Free PMC article.
Temporal Trends of Wild-Type Transthyretin Amyloid Cardiomyopathy in the Transthyretin Amyloidosis Outcomes Survey.
Nativi-Nicolau J, Siu A, Dispenzieri A, Maurer MS, Rapezzi C, Kristen AV, Garcia-Pavia P, LoRusso S, Waddington-Cruz M, Lairez O, Witteles R, Chapman D, Amass L, Grogan M; THAOS Investigators. Nativi-Nicolau J, et al. JACC CardioOncol. 2021 Oct 19;3(4):537-546. doi: 10.1016/j.jaccao.2021.08.009. eCollection 2021 Oct. JACC CardioOncol. 2021. PMID: 34729526 Free PMC article.
Phase 2 Trial of Rituximab in Acetylcholine Receptor Antibody-Positive Generalized Myasthenia Gravis: The BeatMG Study.
Nowak RJ, Coffey CS, Goldstein JM, Dimachkie MM, Benatar M, Kissel JT, Wolfe GI, Burns TM, Freimer ML, Nations S, Granit V, Smith AG, Richman DP, Ciafaloni E, Al-Lozi MT, Sams LA, Quan D, Ubogu E, Pearson B, Sharma A, Yankey JW, Uribe L, Shy M, Amato AA, Conwit R, O'Connor KC, Hafler DA, Cudkowicz ME, Barohn RJ; NeuroNEXT NN103 BeatMG Study Team. Nowak RJ, et al. Among authors: quan d. Neurology. 2022 Jan 25;98(4):e376-e389. doi: 10.1212/WNL.0000000000013121. Neurology. 2022. PMID: 34857535 Free PMC article.
Clinical and genetic profile of patients enrolled in the Transthyretin Amyloidosis Outcomes Survey (THAOS): 14-year update.
Dispenzieri A, Coelho T, Conceição I, Waddington-Cruz M, Wixner J, Kristen AV, Rapezzi C, Planté-Bordeneuve V, Gonzalez-Moreno J, Maurer MS, Grogan M, Chapman D, Amass L; THAOS investigators. Dispenzieri A, et al. Orphanet J Rare Dis. 2022 Jun 18;17(1):236. doi: 10.1186/s13023-022-02359-w. Orphanet J Rare Dis. 2022. PMID: 35717381 Free PMC article.
499 results