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Year Number of Results
1984 1
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52 results

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Page 1
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. 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 Free article. Clinical Trial.
Five-Year Results With Patisiran for Hereditary Transthyretin Amyloidosis With Polyneuropathy: A Randomized Clinical Trial With Open-Label Extension.
Adams D, Wixner J, Polydefkis M, Berk JL, Conceição IM, Dispenzieri A, Peltier A, Ueda M, Bender S, Capocelli K, Jay PY, Yureneva E, Obici L; patisiran Global OLE study group. Adams D, et al. JAMA Neurol. 2025 Mar 1;82(3):228-236. doi: 10.1001/jamaneurol.2024.4631. JAMA Neurol. 2025. PMID: 39804640 Free PMC article. Clinical Trial.
Multidisciplinary approach in the management of hATTR.
Losada I, González-Moreno J, Rodriguez A, Uson M, Ripoll-Vera T, Ferrer-Nadal A, Rigo E, Andreu H, Figuerola A, Montalà JC, Descals C, Álvarez J, Vega-Mañés F, Roig A, Cisneros-Barroso E. Losada I, et al. Among authors: uson m. Eur J Clin Invest. 2020 Oct;50(10):e13296. doi: 10.1111/eci.13296. Epub 2020 Aug 11. Eur J Clin Invest. 2020. PMID: 32474919 No abstract available.
Significant reduction in proteinuria after treatment with tafamidis.
Ferrer-Nadal A, Ripoll T, Uson M, Figuerola A, Andreu H, Losada I, Gonzalez J, Cisneros-Barroso E, Buades J. Ferrer-Nadal A, et al. Among authors: uson m. Amyloid. 2019;26(sup1):67-68. doi: 10.1080/13506129.2019.1583186. Amyloid. 2019. PMID: 31343359 No abstract available.
PDGF-BB serum levels are decreased in adult onset Pompe patients.
Fernández-Simón E, Carrasco-Rozas A, Gallardo E, Figueroa-Bonaparte S, Belmonte I, Pedrosa I, Montiel E, Suárez-Calvet X, Alonso-Pérez J, Segovia S, Nuñez-Peralta C, Llauger J, Mayos M, Illa I; Spanish Pompe Study Group; Díaz-Manera J. Fernández-Simón E, et al. Sci Rep. 2019 Feb 14;9(1):2139. doi: 10.1038/s41598-018-38025-0. Sci Rep. 2019. PMID: 30765719 Free PMC article.
Identification of serum microRNAs as potential biomarkers in Pompe disease.
Carrasco-Rozas A, Fernández-Simón E, Lleixà MC, Belmonte I, Pedrosa-Hernandez I, Montiel-Morillo E, Nuñez-Peralta C, Llauger Rossello J, Segovia S, De Luna N, Suarez-Calvet X, Illa I; Pompe Spanish Study group; Díaz-Manera J, Gallardo E. Carrasco-Rozas A, et al. Ann Clin Transl Neurol. 2019 Jul;6(7):1214-1224. doi: 10.1002/acn3.50800. Epub 2019 Jun 12. Ann Clin Transl Neurol. 2019. PMID: 31353854 Free PMC article. Clinical Trial.
Quantitative muscle MRI to follow up late onset Pompe patients: a prospective study.
Figueroa-Bonaparte S, Llauger J, Segovia S, Belmonte I, Pedrosa I, Montiel E, Montesinos P, Sánchez-González J, Alonso-Jiménez A, Gallardo E, Illa I; Spanish Pompe group; Díaz-Manera J. Figueroa-Bonaparte S, et al. Sci Rep. 2018 Jul 18;8(1):10898. doi: 10.1038/s41598-018-29170-7. Sci Rep. 2018. PMID: 30022036 Free PMC article.
Validating the usefulness of Sudoscan in ATTRv: a single centre experience.
Moreno-Moraleda E, González-Moreno J, Cisneros-Barroso E, Ribot-Sansó MA, Ripoll-Vera T, Descals C, Uson M, Montalà JC, Figuerola A, Rodríguez A, Losada I. Moreno-Moraleda E, et al. Among authors: uson m. Neurol Sci. 2024 Jun;45(6):2859-2867. doi: 10.1007/s10072-024-07311-3. Epub 2024 Jan 23. Neurol Sci. 2024. PMID: 38261153
52 results