Zobrazeno 1 - 10
of 94
pro vyhledávání: '"Tina Duong"'
Autor:
Thomas O. Crawford, John W. Day, Darryl C. De Vivo, Jena M. Krueger, Eugenio Mercuri, Andres Nascimento, Amy Pasternak, Elena Stacy Mazzone, Tina Duong, Guochen Song, Jing L. Marantz, Scott Baver, Dongzi Yu, Lan Liu, Basil T. Darras
Publikováno v:
Frontiers in Neurology, Vol 15 (2024)
Background and purposeAt 12 months in the phase 2 TOPAZ study, treatment with apitegromab was associated with both an improved motor function in patients with Type 2 or 3 spinal muscular atrophy (SMA) and with a favorable safety profile. This manuscr
Externí odkaz:
https://doaj.org/article/463093cc41344076b301b431a80c1691
Autor:
Charlotte Lilien, Eva Vrscaj, Gita Thapaliya, Nicolas Deconinck, Liesbeth De Waele, Tina Duong, Jana Haberlová, Markéta Kumhera, Geertrui Peirens, Lena Szabo, Valentine Tahon, Whitney J. Tang, Noor Benmhammed, Laurie Médard, Laurent Servais
Publikováno v:
Journal of Clinical Medicine, Vol 13, Iss 12, p 3418 (2024)
Background and Objective: Patients with spinal muscular atrophy (SMA) treated with a disease-modifying therapy (DMT) are often classified as responders or non-responders based on the attainment of a specific improvement threshold on validated functio
Externí odkaz:
https://doaj.org/article/b00a494feb964e37a29264d9282d385e
Autor:
Claudia A. Chiriboga, Claudio Bruno, Tina Duong, Dirk Fischer, Eugenio Mercuri, Janbernd Kirschner, Anna Kostera-Pruszczyk, Birgit Jaber, Ksenija Gorni, Heidemarie Kletzl, Imogen Carruthers, Carmen Martin, Francis Warren, Renata S. Scalco, Kathryn R. Wagner, Francesco Muntoni, the JEWELFISH Study Group
Publikováno v:
Neurology and Therapy, Vol 12, Iss 5, Pp 1799-1801 (2023)
Externí odkaz:
https://doaj.org/article/258bc786e54b4365b79dbe9dd14b5d86
Autor:
Claudia A. Chiriboga, Claudio Bruno, Tina Duong, Dirk Fischer, Eugenio Mercuri, Janbernd Kirschner, Anna Kostera-Pruszczyk, Birgit Jaber, Ksenija Gorni, Heidemarie Kletzl, Imogen Carruthers, Carmen Martin, Francis Warren, Renata S. Scalco, Kathryn R. Wagner, Francesco Muntoni, the JEWELFISH Study Group
Publikováno v:
Neurology and Therapy, Vol 12, Iss 2, Pp 543-557 (2023)
Abstract Introduction Risdiplam is a survival of motor neuron 2 (SMN2) splicing modifier for the treatment of patients with spinal muscular atrophy (SMA). The JEWELFISH study (NCT03032172) was designed to assess the safety, tolerability, pharmacokine
Externí odkaz:
https://doaj.org/article/cf9aa173befd4087b4e4bc7cce3e9e84
Autor:
Whitney J. Tang, Bo Gu, Samuel Montalvo, Sally Dunaway Young, Dana M. Parker, Constance de Monts, Paxton Ataide, Noirin Ni Ghiollagain, Matthew T. Wheeler, Carolina Tesi Rocha, Jeffrey W. Christle, Zihuai He, John W. Day, Tina Duong
Publikováno v:
Journal of Clinical Medicine, Vol 12, Iss 24, p 7582 (2023)
Assessing endurance in non-ambulatory individuals with Spinal Muscular Atrophy (SMA) has been challenging due to limited evaluation tools. The Assisted 6-Minute Cycling Test (A6MCT) is an upper limb ergometer assessment used in other neurologic disor
Externí odkaz:
https://doaj.org/article/d7a56aefb4154cbcbb6ed794fe26bf48
Autor:
Marie Beaudin, Tahereh Kamali, Whitney Tang, Katharine A. Hagerman, Sally Dunaway Young, Lisa Ghiglieri, Dana M. Parker, Benoit Lehallier, Carolina Tesi-Rocha, Jacinda B. Sampson, Tina Duong, John W. Day
Publikováno v:
Journal of Clinical Medicine, Vol 12, Iss 20, p 6696 (2023)
Disease-modifying treatments have transformed the natural history of spinal muscular atrophy (SMA), but the cellular pathways altered by SMN restoration remain undefined and biomarkers cannot yet precisely predict treatment response. We performed an
Externí odkaz:
https://doaj.org/article/eb188060752c483a9a4981a865ea25fe
Autor:
Tina Duong, Jessica Braid, Hannah Staunton, Aurelie Barriere, Fani Petridis, Johannes Reithinger, Rosangel Cruz, Jill Jarecki, Mencia De Lemus, Nicole Gusset, Ria Broekgaarden, Sharan Randhawa, Jessica Flynn, Rob Arbuckle, Sonia Reif, Lida Yang, Angela De Martini, Carole Vuillerot
Publikováno v:
BMC Neurology, Vol 21, Iss 1, Pp 1-4 (2021)
Externí odkaz:
https://doaj.org/article/3e7969a63e0b479d964476d929ecea58
Autor:
Maria Carmela Pera, Giorgia Coratti, Francesca Bovis, Marika Pane, Amy Pasternak, Jacqueline Montes, Valeria A. Sansone, Sally Dunaway Young, Tina Duong, Sonia Messina, Irene Mizzoni, Adele D’Amico, Matthew Civitello, Allan M. Glanzman, Claudio Bruno, Francesca Salmin, Simone Morando, Roberto De Sanctis, Maria Sframeli, Laura Antonaci, Anna Lia Frongia, Annemarie Rohwer, Mariacristina Scoto, Darryl C. De Vivo, Basil T. Darras, John Day, William Martens, Katia A. Patanella, Enrico Bertini, Francesco Muntoni, Richard Finkel, Eugenio Mercuri, the iSMAC group
Publikováno v:
Annals of Clinical and Translational Neurology, Vol 8, Iss 8, Pp 1622-1634 (2021)
Abstract Objective We report longitudinal data from 144 type III SMA pediatric and adult patients treated with nusinersen as part of an international effort. Methods Patients were assessed using Hammersmith Functional Motor Scale Expanded (HFMSE), Re
Externí odkaz:
https://doaj.org/article/934d5becf2b645da80440f5df9f6f4de
Autor:
Tina Duong, Jessica Braid, Hannah Staunton, Aurelie Barriere, Fani Petridis, Johannes Reithinger, Rosangel Cruz, Jill Jarecki, Mencia De Lemus, Nicole Gusset, Ria Broekgaarden, Sharan Randhawa, Jessica Flynn, Rob Arbuckle, Sonia Reif, Lida Yang, Angela De Martini, Carole Vuillerot
Publikováno v:
BMC Neurology, Vol 21, Iss 1, Pp 1-14 (2021)
Abstract Background The 32-item Motor Function Measure (MFM32) is a clinician-reported outcome measure used to assess the functional abilities of individuals with neuromuscular diseases, including those with spinal muscular atrophy (SMA). This two-pa
Externí odkaz:
https://doaj.org/article/40a4d4e72db647e4b617a358a6c10774
Autor:
Anna G. Mayhew, Meredith K. James, Ursula Moore, Helen Sutherland, Marni Jacobs, Jia Feng, Linda Pax Lowes, Lindsay N. Alfano, Robert Muni Lofra, Laura E. Rufibach, Kristy Rose, Tina Duong, Luca Bello, Irene Pedrosa-Hernández, Scott Holsten, Chikako Sakamoto, Aurélie Canal, Nieves Sánchez-Aguilera Práxedes, Simone Thiele, Catherine Siener, Bruno Vandevelde, Brittney DeWolf, Elke Maron, Heather Gordish-Dressman, Heather Hilsden, Michela Guglieri, Jean-Yves Hogrel, Andrew M. Blamire, Pierre G. Carlier, Simone Spuler, John W. Day, Kristi J. Jones, Diana X. Bharucha-Goebel, Emmanuelle Salort-Campana, Alan Pestronk, Maggie C. Walter, Carmen Paradas, Tanya Stojkovic, Madoka Mori-Yoshimura, Elena Bravver, Jordi Díaz-Manera, Elena Pegoraro, Jerry R. Mendell, Volker Straub
Publikováno v:
Frontiers in Neurology, Vol 13 (2022)
Dysferlinopathy is a muscular dystrophy with a highly variable functional disease progression in which the relationship of function to some patient reported outcome measures (PROMs) has not been previously reported. This analysis aims to identify the
Externí odkaz:
https://doaj.org/article/0470816544d14c339d5608df7267ca17