Zobrazeno 1 - 10
of 178
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:
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:
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:
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
Autor:
Tina Duong, Hannah Staunton, Jessica Braid, Aurelie Barriere, Ben Trzaskoma, Ling Gao, Tom Willgoss, Rosangel Cruz, Nicole Gusset, Ksenija Gorni, Sharan Randhawa, Lida Yang, Carole Vuillerot
Publikováno v:
Frontiers in Neurology, Vol 12 (2022)
The 32-item Motor Function Measure (MFM32) is an assessment of motor function used to evaluate fine and gross motor ability in patients with neuromuscular disorders, including spinal muscular atrophy (SMA). Reliability and validity of the MFM32 have
Externí odkaz:
https://doaj.org/article/bfda0d067a5e46119881b84dee5d1662
Autor:
Tina Duong, Kristin J. Krosschell, Meredith K. James, Leslie Nelson, Lindsay N. Alfano, Katy Eichinger, Elena Mazzone, Kristy Rose, Linda P. Lowes, Anna Mayhew, Julaine Florence, Wendy King, Claudia R. Senesac, Michelle Eagle
Publikováno v:
Frontiers in Genetics, Vol 12 (2021)
Critical components of successful evaluation of clinical outcome assessments (COAs) in multisite clinical trials and clinical practice are standardized training, administration, and documented reliability of scoring. Experiences of evaluators, alongs
Externí odkaz:
https://doaj.org/article/981e516b28d04a8384a87ee77f7b25aa
Autor:
Sally Dunaway Young, Amy Pasternak, Tina Duong, Katlyn E. McGrattan, Sarah Stranberg, Elizabeth Maczek, Courtney Dias, Whitney Tang, Dana Parker, Alexis Levine, Alyssa Rohan, Connie Wolford, William Martens, Michael P. McDermott, Basil T. Darras, John W. Day
Publikováno v:
Journal of Neuromuscular Diseases. 10:199-209
Background: Novel Spinal Muscular Atrophy (SMA) treatments have demonstrated improvements on motor measures that are clearly distinct from the natural history of progressive decline. Comparable measures are needed to monitor bulbar function, which is