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. CuATSM effectively ameliorates ALS patient astrocyte‐mediated motor neuron toxicity in human in vitro models of amyotrophic lateral sclerosis. Issue 2 (10th October 2022) Authors: Dennys, Cassandra N.; Roussel, Florence; Rodrigo, Rochelle; Zhang, Xiaojin; Sierra Delgado, Andrea; Hartlaub, Annalisa; Saelim‐Ector, Asya; Ray, Will; Heintzman, Sarah; Fox, Ashley; Kolb, Stephen J.; Beckman, Joseph; Franco, Maria Clara; Meyer, Kathrin Journal: Glia Issue: Volume 71:Issue 2(2023) Page Start: 350 Record Type: Journal Article View Content: Available online (eLD content is only available in our Reading Rooms) ↗
3. 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) ↗
4. Psychometric evaluation of modified spinal muscular atrophy functional rating scale (SMAFRS) in adult patients using Rasch analysis. Issue 3 (15th January 2023) Authors: Sadjadi, Reza; Kelly, Kristina; Glanzman, Allan M.; Montes, Jacqueline; Linsenmayer, Matthew; Tellez, Marco; Bartlett, Amy; Heintzman, Sarah; Kolb, Stephen J.; Arnold, W. David; El‐Sheikh, Bakri Journal: Muscle & nerve Issue: Volume 67:Issue 3(2023) Page Start: 239 Record Type: Journal Article View Content: Available online (eLD content is only available in our Reading Rooms) ↗
5. Safety and efficacy of zilucoplan in patients with generalised myasthenia gravis (RAISE): a randomised, double-blind, placebo-controlled, phase 3 study. Issue 5 (May 2023) Authors: Howard, James F; Bresch, Saskia; Genge, Angela; Hewamadduma, Channa; Hinton, John; Hussain, Yessar; Juntas-Morales, Raul; Kaminski, Henry J; Maniaol, Angelina; Mantegazza, Renato; Masuda, Masayuki; Sivakumar, Kumaraswamy; Śmiłowski, Marek; Utsugisawa, Kimiaki; Vu, Tuan; Weiss, Michael D; Zajda, M... Journal: Lancet neurology Issue: Volume 22:Issue 5(2023) Page Start: 395 Record Type: Journal Article View Content: Available online (eLD content is only available in our Reading Rooms) ↗
6. Persistent neuromuscular junction transmission defects in adults with spinal muscular atrophy treated with nusinersen. Issue 2 (12th August 2021) Authors: Arnold, W David; Severyn, Steven; Zhao, Songzhu; Kline, David; Linsenmayer, Matthew; Kelly, Kristina; Tellez, Marco; Bartlett, Amy; Heintzman, Sarah; Reynolds, Jerry; Sterling, Gary; Weaver, Tristan; Rajneesh, Kiran; Burghes, Arthur H M; Kolb, Stephen J; Elsheikh, Bakri Journal: BMJ neurology open Issue: Volume 3:Issue 2(2021) Page Start: Record Type: Journal Article View Content: Available online (eLD content is only available in our Reading Rooms) ↗