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Browsing by Author "Franco, Ana"

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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
    Publication . Adams, David; Polydefkis, Michael; González-Duarte, Alejandra; Wixner, Jonas; Kristen, Arnt V.; Schmidt, Hartmut H.; Berk, John L.; Losada López, Inés Asunción; Dispenzieri, Angela; Quan, Dianna; Conceição, Isabel M.; Kyriakides, Theodoros; Waucquier, Nawal; Campana-Salort, Emmanuelle; Wiesman, Janice; Zueiter, Andreas; Rezk, Tamer; Perez, Javier M.; Castro, Isabel; Cardoso, Marcio; Ryder, Julie; Théaudin-Saliou, Marie; Zamba-Papanicolaou, Eleni; Kraus, Deborah; Kim, Byoung-Joon; Correia, Nuno; Min, Ju-Hong; Franco, Ana; Suhr, Ole; Katanazaka, Kimitaka; Taylor, Mark; Hayman, Suzanne R.; Agathangelou, Petros; Aldinc, Emre; Coskun, Derya J.; Diallo, Saran; Dias da Silva, Moises; Suan, Daniel; Dalton, James; Yang, Chih-Chao; Viera Lima, Ana Flavia; Mezei, Michelle M.; Brice, Sylvie; Sellers, Peter; Abu-Manneh, Rana; Cortese, Andrea; Leonidou, Eleni; Stone, Paula; Hobbs, Miriam; Cauquil-Michon, Cécile; Ehinger, Vincent; Balaya-Gouraya, Line; Boyer, Leslie; Seok, Jin Myoung; Signate, Aissatou; Khoshnoodi, Mohammad; Criollo Mora, Elia; Jozefowicz, Elsa; Wajnsztajn, Fernanda; Taniguchi, Ikuko; Eliahou, Ludivine; Pouget, Jean; Oda, Junko; Soderberg, Karin; Le Corvoisier, Philippe; Nienhuis, Hans; Chaudhry, Vinay; Victory, Jennifer; Yamashita, Taro; Rezzan, Kus Jülide; Guerrero Sola, Antonio; Luigetti, Marco; Dalia, Samir; Ajroud-Driss, Senda Ajroud-Driss; Kenepp, Amanda; Bournane, Halima; Hwa, Lisa; Segawa, Akiko; Martins da Silva, Ana; Kapoor, Prashant; Liu, Qingfeng; Hokezu, Youichi; Gorevic, Peter; Pinto, Luiz Felipe; Palacios, Marta; Lambrianides, Sakis; Berthelot, Emeline; Karamursel, Sacit; Tugal Tutkun, Nuriye Ilknur; Kasprzyk, Emmanuelle; Elrezzi, Elise; Gonzalez, Leidy J.; Verschueren, Annie; Sweetser, Marianne T.; García Feijoo, Julián; Rosti, Vittorio; Lee, Kia; Choi, Kyomin; Anan, Intissar; Mihailovic, Natasa; Oshita, Tomohiko; Rajkumar, Vincent S.; Pedrosa-Domellöf, Fatima; Hipólito Reis, Antonio; Koujin, Yoko; Walter, Doreen; Hosokawa, Yuko; Meguig, Sayah; Vista, Geno; Lozza, Alessandro; Hachulla, Eric; Lee, Hye Lim; Gauvain, Clement; Yeh, Shin-Joe; Martin, Nathalie; Dioso, Holly; Chu, Hanh; Özdemir, Betül; Frenzel, Lukas F.; Pytel Cordoba, Vanesa; Cherneva, Zheyna; Chacko, Liza; Tran, Leyla; Munoz Beamud, Francisco; Attarian, Shahram; Elabed, Mongia; Marques Júnior, Wilson; Pester, Lars; Gertz, Morie A.; Lazzari, Victoria; Hein, Selina; Orellana, Lucas G.; Stewart, Graeme; Sacko, Niamey; Pérez-Castañeda, Sandra Lorena; Marques Coutinho, Barbara; Coelho, Teresa; Lopate, Glenn; Ruiz, Julie; Sakurada, Naonori; Do, Thi Hoa (Therese); Gervais de Santa Rosa, Renata; Tournev, Ivaylo; Serbest, Nail G.; Freitas, Monica; Rodas Marin, Lida Maria; Nordh, Erik; Dyck, Peter; Mazzeo, Anna; Lin, Yi; DeLong, Douglas; Ernande, Laura; Vervoitte, Laetitia; Chan, Soo Looi; Zibert, Andree; Meng, Ulrike; Lee, Ming-Jen; Shah, Syed Mahmood; Yamanishi, Yuki; Miyazawa, Chinatsu; Zhelyazkova-Glaveeva, Sashka; Fujita, Akemi; Hsieh, Sung-Tsang; Gerk, Olga; Lee, Ga Yeon; Dingli, David; Vuong, Thao; Russo, Massimo; Hrkman, Nicole; Matsumoto, Kazuhiko; Miller, Sarah; Nguyen, David; Smith, Ashley; Rigo Oliver, Elena; Moreira, Carolina Lavigne; Martinez-Naharro, Ana; Takahashi, Tetsuya; Berber, Erhan; Low, Soon Chai; Koto, Shusuke; Shimomura, Masahiro; Itoya, Amenze; Ulbricht, Hannah; Ferreira, Natalia; Go, Ronald; Jarrett, Raquel; Cho, Eun Bin; Pu, Helen; Florence, Julaine; Algalarrondo, Vincent; Bernardo, Daniela; Li, Steve; Oda, Tetsuya; Taylor, Lenka; Vest, John; Jehan Lacour, Nathalie; Zepeda, Karla A.; Rakotondratafika, Fetra; Ye, Jing; Scheid, Christof; Morino, Hiroyuki; Overcash, J. Scott; Lachmann, Helen J.; Campistol, Josep M.; Cantú Brito, Carlos Gerardo; White, Matthew T.; O'Riordan, William; Inamo, Jocelyn; Paras, Allan; Sarafov, Staiko; Lahme, Larissa; Casey, Patricia; Heintzman, Sarah; Pinto, Marcus Vinicius; Vinas, Maita; Stephant, Maeva; Giang, Lien; Lin, Kon-Ping; Leung, Nelson; Lopes, Ana; Bisogni, Giulia; Kim, Arreum; Obici, Laura; Cárdenas-Soto, Karla; Friebel, Frauke; Monte, Julia R.; Litcanov, Debora Cristina; Bulthuis-Kuiper, Janita; Cohen, Oliver; Gamez Martinez, Jose M; Fukase, Emi; Brannagan III, Thomas H.; Gillmore, Julian D.; Descals, Cristina; Dávila, Lucía Galán; Cisneros Barroso, Eugenia; Truong, Nghi; Uson, Mercedes; Gonsalves, Wilson; Jose Vega, Francisco; Charlin, Cyril; Moritaka, Taiga; Huesing-Kabar, Anna; Mason, Garnette; Pereira, Pedro; Castro, Jose; Masuda, Teruaki; Sommerville, R. Brian; Figuerola, Antoni; Lust, John A; Chao, Chi-Chao; Mauricio, Elizabeth A.; Tard, Celine; Holtick, Udo; Romano, Angela; Kim, JuHyeon; Carrillo, Esmeralda; aus dem Siepen, Fabian; Lee, Yi-Chung; Not, Adeline; Giardino, Elena; Becquemont, Laurent; Kumar, Shaji K.; Hernandez Reyes, Jose Pablo; Sankey, Alison; Jack, Kristin; Barroso, Fabio; LoRusso, Samantha; Gaouar, Amina; Baskin, Amber; Lamas, Filipa; Lautre, Andrea; Schilling, Matthias; Rodríguez Rodríguez, Adrián; Chamova, Teodora; Wang, Jing Jing; Pessoa, Bernardete; Misumi, Yohei; Guimaraes, Nadia; Ueno, Hiroki; Dixon, Stacy; Waters, Michael; Law, Steven; Weimer, Louis; Deplanque, Dominique; Noe-Schwenn, Simone; Dimberg, Elliot; Cakar, Arman; Russell, Stephen J.; Shin, Hyun Jin; Beaudonnet, Guillemette; Harada, Masaya; Burks, Kathleen L.; Dang, Lien; Blume, Brianna; Tsang, Steven; Oh, Jeeyoung; Delmont, Emilien; Nezu, Tomohisa; Ueda, Akihiko; Wells, Karen; Alves, Cristina; Nakahara, Keiichi; Yorita, Akiko; Ando, Rina; González Moreno, Juan; Di Paolantonio, Andrea; Kishida, Dai; Tsuruhisa, Seiko; Elitok, Ali; Migaud-Chervy, Maria-Claire; Witzig, Thomas E.; Cioroiu, Comana; Arrouasse, Raphaele; Perez de Isla, Leopoldo; Allen, Adriane C.; Jeon, Eun-Seok; Schubert, Friederike; Nakano, Takahiro; Falk, Malin; Daccach Marques, Vanessa; Kleyman, Inna; Cherninkova-Gopina, Sylvia; Taniwaki, Takayuki; Planté-Bordeneuve, Violaine; Fayerman, Raisy; Tada, Satoshi; Endo, Hironobu; Montala, Carles; Lebellec, Loic; Guglielmino, Valeria; del Mar Saniger Alba, Maria; Salhi, Hayet; Cardoso Berensztejn, Amanda; Falcao de Campos, Catarina; Rougeaux, Isabelle; Le, An; Ayache, Samar S.; Kato, Nagaaki; Zwijack, Brittany; Gentile, Luca; Lindqvist, Per; Kuzume, Daisuke; Judge, Daniel P.; Sabatelli, Mario; Grapperon, Aude-Marie; Ueda, Mitsuharu; Fonder, Amie; Al-Salameh, Abdallah; Lecoq, Anne-Lise; Blasco Pelicano, Josep Miquel; Sirin Inan, Nermin G.; Labeyrie, Celine; Gragera Martínez, Álvaro; Imai, Akira; Brueckner, Caitlin; Su, Jen-Jen; Gilmore, Catherine; Meyrath, David; Gendre, Thierry; Hineno, Akiyo; Chahine, Ilonah; Boubrit, Yasmine; Kodaira, Minori; Yoshinaga, Tsuneaki; Shije, Jeffrey; Slama, Michel S.; Sekijima, Yoshiki; Rivera de la Parra, David; Nagai, Masahiro; Neves, Carlos; Moscardini, Mariana; Park, Jae Hong; Horga, Alejandro; Choumert, Ariane; Lim, He Eng Li; Valdrez, Katia; Miyahara, Teruyoshi; Wade, N. Kevin; Kim, Hee Jin; Thai, Desmond; de Azevedo Coutinho, Conceição; Bauer, Ralf; Ramalho, Joana; May, John; Joslin, Benjamin C.; Santos, Miguel Oliveira; Freimer, Miriam; Ulane, Christina; Megelin, Thomas; Pedrosa, Salve; Pilebro, Bjorn; Borrachero, Cristina; Sanz, Monica; Owusu-Sekyere, Mercy; Nguyen, Tyler; Echaniz-Laguna, Andoni; Altinkurt, Emre; Rodrigues, Leonardo; Zeldenrust, Steven R; Tsai, Li-Kai; Kabar, Iyad; Hamaguchi, Hirotoshi; Jiménez López, Brenda Poled; Green, Sinikka; Vorona, Elena; Waddington-Cruz, Márcia; Merlini, Giampaolo; Durmus, Hacer; Neshige, Shuichiro; Meyle, Eva; Goh, Khean Jin; Sahin, Erdi; Kleinschmidt, Natalia; Focseneanu, Cecile; Parman, Yesim; Rousseau, Antoine; Mekhael, Linda
    Background: Hereditary transthyretin-mediated amyloidosis is a rare, inherited, progressive disease caused by mutations in the transthyretin (TTR) gene. We assessed the safety and efficacy of long-term treatment with patisiran, an RNA interference therapeutic that inhibits TTR production, in patients with hereditary transthyretin-mediated amyloidosis with polyneuropathy. Methods: This multicentre, open-label extension (OLE) trial enrolled patients at 43 hospitals or clinical centres in 19 countries as of Sept 24, 2018. Patients were eligible if they had completed the phase 3 APOLLO or phase 2 OLE parent studies and tolerated the study drug. Eligible patients from APOLLO (patisiran and placebo groups) and the phase 2 OLE (patisiran group) studies enrolled in this global OLE trial and received patisiran 0·3 mg/kg by intravenous infusion every 3 weeks with plans to continue to do so for up to 5 years. Efficacy assessments included measures of polyneuropathy (modified Neuropathy Impairment Score +7 [mNIS+7]), quality of life, autonomic symptoms, nutritional status, disability, ambulation status, motor function, and cardiac stress, with analysis by study groups (APOLLO-placebo, APOLLO-patisiran, phase 2 OLE patisiran) based on allocation in the parent trial. The global OLE is ongoing with no new enrolment, and current findings are based on the interim analysis of the patients who had completed 12-month efficacy assessments as of the data cutoff. Safety analyses included all patients who received one or more dose of patisiran up to the data cutoff. This study is registered with ClinicalTrials.gov, NCT02510261. Findings: Between July 13, 2015, and Aug 21, 2017, of 212 eligible patients, 211 were enrolled: 137 patients from the APOLLO-patisiran group, 49 from the APOLLO-placebo group, and 25 from the phase 2 OLE patisiran group. At the data cutoff on Sept 24, 2018, 126 (92%) of 137 patients from the APOLLO-patisiran group, 38 (78%) of 49 from the APOLLO-placebo group, and 25 (100%) of 25 from the phase 2 OLE patisiran group had completed 12-month assessments. At 12 months, improvements in mNIS+7 with patisiran were sustained from parent study baseline with treatment in the global OLE (APOLLO-patisiran mean change -4·0, 95 % CI -7·7 to -0·3; phase 2 OLE patisiran -4·7, -11·9 to 2·4). Mean mNIS+7 score improved from global OLE enrolment in the APOLLO-placebo group (mean change from global OLE enrolment -1·4, 95% CI -6·2 to 3·5). Overall, 204 (97%) of 211 patients reported adverse events, 82 (39%) reported serious adverse events, and there were 23 (11%) deaths. Serious adverse events were more frequent in the APOLLO-placebo group (28 [57%] of 49) than in the APOLLO-patisiran (48 [35%] of 137) or phase 2 OLE patisiran (six [24%] of 25) groups. The most common treatment-related adverse event was mild or moderate infusion-related reactions. The frequency of deaths in the global OLE was higher in the APOLLO-placebo group (13 [27%] of 49), who had a higher disease burden than the APOLLO-patisiran (ten [7%] of 137) and phase 2 OLE patisiran (0 of 25) groups. Interpretation: In this interim 12-month analysis of the ongoing global OLE study, patisiran appeared to maintain efficacy with an acceptable safety profile in patients with hereditary transthyretin-mediated amyloidosis with polyneuropathy. Continued long-term follow-up will be important for the overall assessment of safety and efficacy with patisiran.
    2021Journal article Restricted access Show more
  • Modelo económico bancário : o caso português
    Publication . Barata, José Martins; Franco, Ana; Marçal, António; Cêrcas, Francisco; Barata, Luís
    Este trabalho constitui uma primeira tentativa de experimentação de um modelo económico de previsão bancária, no contexto institucional português. O objectivo proposto consiste, por conseguinte, na apresentação de uma fundamentação teórica para o planeamento na · Banca portuguesa, de modo a proporcionar-se a obtenção de ensinamentos aplicáveis a casos concretos. Tratando-se da conciliação de duas áreas normalmente separadas no domínio académico - a macro e a microeconomia - num campo tão especializado como é o da actividade bancária, deverá reconhecer-se que o objectivo visado é ambicioso, quer por razões de natureza científica, quer por motivos relacionados com a disponibilidade de informação.
    1984Working paper Open access Show more
  • Zona Franco e sistema bancário africano
    Publication . Franco, Ana
    A Zona Franco, ou mais correctamente, as duas sub-zonas franco começaram por ser um espaço geograficamente definido ·entre a França ·e as suas colónias africanas onde. se procedia livremente as transferências comerciais e financeiras. Depois de 1966, com a liberalização dos fluxos financeiros entre a França e os restantes países, a caracterização das relações entre a França e as suas ex-colónias adquiriu um significado marcadamente monetário. Hoje a França define com seis países da África Central (Camarões, Congo, Gabão, República Centro Africana, Tchad e Guiné· Equatorial) e com sete países ·da África Ocidental (Costa do Marfim, Benin, Burkina, Faro, Niger, Senegal, Togol e Mali) uma zona monetária (a Zona Franco) cuja característica determinante é a convenção estabelecida entre o Banco Emissor destes Estados e o Tesouro francês, de constituição de uma “conta de operações". A existência desta "conta de operações” permite a convertibilidade das moedas africanas em francos franceses no interior da Zona Franco (a uma taxa de câmbio fixa), assim como a gestão comum das reservas de ouro e divisas.
    1988-09Working paper Open access Show more