1. Long-term safety and efficacy of patisiran for hereditary transthyretin-mediated amyloidosis with polyneuropathy: 12-month results of an open-label extension study. Issue 1 (January 2021) Authors: 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; Slama, Michel S; Gillmore, Julian D; Kyriakides, Theodoros; Ajroud-Driss, Senda; Wa... Journal: Lancet neurology Issue: Volume 20:Issue 1(2021) Page Start: 49 Record Type: Journal Article View Content: Available online (eLD content is only available in our Reading Rooms) ↗
2. Long-term safety and efficacy of patisiran for hereditary transthyretin-mediated amyloidosis with polyneuropathy: 12-month results of an open-label extension study. Issue 1 (January 2021) Authors: 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; Slama, Michel S; Gillmore, Julian D; Kyriakides, Theodoros; Ajroud-Driss, Senda; Wa... Journal: Lancet neurology Issue: Volume 20:Issue 1(2021) Page Start: 49 Record Type: Journal Article View Content: Available online (eLD content is only available in our Reading Rooms) ↗
3. Regulator of telomere length 1 (RTEL1) mutations are associated with heterogeneous pulmonary and extra-pulmonary phenotypes. Issue 2 (7th February 2019) Authors: Borie, Raphael; Bouvry, Diane; Cottin, Vincent; Gauvain, Clement; Cazes, Aurélie; Debray, Marie-Pierre; Cadranel, Jacques; Dieude, Philippe; Degot, Tristan; Dominique, Stephane; Gamez, Anne Sophie; Jaillet, Madeleine; Juge, Pierre-Antoine; Londono-Vallejo, Arturo; Mailleux, Arnaud; Mal, Hervé; Bo... Other Names: Callebaut Isabelle author non-byline.; Amar Smail author non-byline.; Magois Eline author non-byline.; Plessier Aurélie author non-byline.; Brillet Pierre-Yves author non-byline.; Kambouchner Marianne author non-byline.; Valeyre Dominique author non-byline.; Uzunhan Yurdagul author non-byline.; D... Journal: European respiratory journal Issue: Volume 53:Issue 2(2019) Page Start: Record Type: Journal Article View Content: Available online (eLD content is only available in our Reading Rooms) ↗