Works by Duong, Tina

Results: 49

Cardiopulmonary exercise testing as an integrative approach to explore physiological limitations in Duchenne muscular dystrophy.

Published in:

Journal of Neuromuscular Diseases, 2025, v. 12, n. 3, p. 408, doi. 10.1177/22143602251319170

By:

Bomma, Meghana;
Lott, Donovan;
Forbes, Sean;
Shih, Renata;
Coppola, John-Anthony;
Christle, Jeffrey W.;
Duong, Tina;
Russo, Joseph;
Pant, Aditi;
Leon-Astudillo, Carmen;
Berthy, Julie;
Cousins, Christina;
Corti, Manuela;
Byrne, Barry;
May, James;
Xue, W.;
Taivassalo, Tanja

Publication type:

Article

Cardiopulmonary Recovery After Maximal Exercise in Individuals with Neuromuscular Disease and Limited Mobility.

Published in:

Journal of Clinical Medicine, 2025, v. 14, n. 12, p. 4190, doi. 10.3390/jcm14124190

By:

Blumberg, Yair;
de Monts, Constance;
Montalvo, Samuel;
Tang, Whitney J.;
Dunaway Young, Sally;
Hageman, Nathan;
Sanchis-Gomar, Fabian;
Ashley, Euan A.;
Amar, David;
Myers, Jonathan;
Wheeler, Matthew T.;
Day, John W.;
Duong, Tina;
Christle, Jeffrey W.

Publication type:

Article

Patients' Perceptions of Nusinersen Effects According to Their Responder Status.

Published in:

Journal of Clinical Medicine, 2024, v. 13, n. 12, p. 3418, doi. 10.3390/jcm13123418

By:

Lilien, Charlotte;
Vrscaj, Eva;
Thapaliya, Gita;
Deconinck, Nicolas;
De Waele, Liesbeth;
Duong, Tina;
Haberlová, Jana;
Kumhera, Markéta;
Peirens, Geertrui;
Szabo, Lena;
Tahon, Valentine;
Tang, Whitney J.;
Benmhammed, Noor;
Médard, Laurie;
Servais, Laurent

Publication type:

Article

Assessing the Assisted Six-Minute Cycling Test as a Measure of Endurance in Non-Ambulatory Patients with Spinal Muscular Atrophy (SMA).

Published in:

Journal of Clinical Medicine, 2023, v. 12, n. 24, p. 7582, doi. 10.3390/jcm12247582

By:

Tang, Whitney J.;
Gu, Bo;
Montalvo, Samuel;
Dunaway Young, Sally;
Parker, Dana M.;
de Monts, Constance;
Ataide, Paxton;
Ni Ghiollagain, Noirin;
Wheeler, Matthew T.;
Tesi Rocha, Carolina;
Christle, Jeffrey W.;
He, Zihuai;
Day, John W.;
Duong, Tina

Publication type:

Article

Cerebrospinal Fluid Proteomic Changes after Nusinersen in Patients with Spinal Muscular Atrophy.

Published in:

Journal of Clinical Medicine, 2023, v. 12, n. 20, p. 6696, doi. 10.3390/jcm12206696

By:

Beaudin, Marie;
Kamali, Tahereh;
Tang, Whitney;
Hagerman, Katharine A.;
Dunaway Young, Sally;
Ghiglieri, Lisa;
Parker, Dana M.;
Lehallier, Benoit;
Tesi-Rocha, Carolina;
Sampson, Jacinda B.;
Duong, Tina;
Day, John W.

Publication type:

Article

Assessing the Relationship of Patient Reported Outcome Measures With Functional Status in Dysferlinopathy: A Rasch Analysis Approach.

Published in:

Frontiers in Neurology, 2022, v. 13, p. 1, doi. 10.3389/fneur.2022.828525

By:

Mayhew, Anna G.;
James, Meredith K.;
Moore, Ursula;
Sutherland, Helen;
Jacobs, Marni;
Jia Feng;
Lowes, Linda Pax;
Alfano, Lindsay N.;
Lofra, Robert Muni;
Rufibach, Laura E.;
Rose, Kristy;
Duong, Tina;
Bello, Luca;
Pedrosa-Hernández, Irene;
Holsten, Scott;
Chikako Sakamoto;
Canal, Aurélie;
Práxedes, Nieves Sánchez-Aguilera;
Thiele, Simone;
Siener, Catherine

Publication type:

Article

A Patient-Centered Evaluation of Meaningful Change on the 32-Item Motor Function Measure in Spinal Muscular Atrophy Using Qualitative and Quantitative Data.

Published in:

Frontiers in Neurology, 2022, p. 1, doi. 10.3389/fneur.2021.770423

By:

Duong, Tina;
Staunton, Hannah;
Braid, Jessica;
Barriere, Aurelie;
Trzaskoma, Ben;
Gao, Ling;
Willgoss, Tom;
Cruz, Rosangel;
Gusset, Nicole;
Gorni, Ksenija;
Randhawa, Sharan;
Yang, Lida;
Vuillerot, Carole

Publication type:

Article

Pulmonary Endpoints in Duchenne Muscular Dystrophy. A Workshop Summary.

Published in:

2017

By:

Finder, Jonathan;
Mayer, Oscar Henry;
Sheehan, Daniel;
Sawnani, Hemant;
Abresch, R. Ted;
Benditt, Joshua;
Birnkrant, David J.;
Duong, Tina;
Henricson, Erik;
Kinnett, Kathi;
McDonald, Craig M.;
Connolly, Anne M.

Publication type:

journal article

Understanding the relationship between the 32-item motor function measure and daily activities from an individual with spinal muscular atrophy and their caregivers' perspective: a two-part study.

Published in:

2021

By:

Duong, Tina;
Braid, Jessica;
Staunton, Hannah;
Barriere, Aurelie;
Petridis, Fani;
Reithinger, Johannes;
Cruz, Rosangel;
Jarecki, Jill;
De Lemus, Mencia;
Gusset, Nicole;
Broekgaarden, Ria;
Randhawa, Sharan;
Flynn, Jessica;
Arbuckle, Rob;
Reif, Sonia;
Yang, Lida;
Martini, Angela De;
Vuillerot, Carole;
De Martini, Angela

Publication type:

journal article

Revised Hammersmith Scale for spinal muscular atrophy: A SMA specific clinical outcome assessment tool.

Published in:

PLoS ONE, 2017, v. 12, n. 2, p. 1, doi. 10.1371/journal.pone.0172346

By:

Ramsey, Danielle;
Scoto, Mariacristina;
Mayhew, Anna;
Main, Marion;
Mazzone, Elena S.;
Montes, Jacqueline;
de Sanctis, Roberto;
Dunaway Young, Sally;
Salazar, Rachel;
Glanzman, Allan M.;
Pasternak, Amy;
Quigley, Janet;
Mirek, Elizabeth;
Duong, Tina;
Gee, Richard;
Civitello, Matthew;
Tennekoon, Gihan;
Pane, Marika;
Pera, Maria Carmela;
Bushby, Kate

Publication type:

Article

Comparison of strength testing modalities in dysferlinopathy.

Published in:

Muscle & Nerve, 2022, v. 66, n. 2, p. 159, doi. 10.1002/mus.27570

By:

Reash, Natalie F.;
James, Meredith K.;
Alfano, Lindsay N.;
Mayhew, Anna G.;
Jacobs, Marni;
Iammarino, Megan A.;
Holsten, Scott;
Sakamoto, Chikako;
Tateishi, Takayuki;
Yajima, Hiroyuki;
Duong, Tina;
de Wolf, Brittney;
Gee, Richard;
Bharucha‐Goebel, Diana X.;
Bravver, Elena;
Mori‐Yoshimura, Madoka;
Bushby, Kate;
Rufibach, Laura E.;
Straub, Volker;
Lowes, Linda P.

Publication type:

Article

Different trajectories in upper limb and gross motor function in spinal muscular atrophy.

Published in:

Muscle & Nerve, 2021, v. 64, n. 5, p. 552, doi. 10.1002/mus.27384

By:

Coratti, Giorgia;
Pera, Maria Carmela;
Montes, Jacqueline;
Pasternak, Amy;
Scoto, Mariacristina;
Baranello, Giovanni;
Messina, Sonia;
Dunaway Young, Sally;
Glanzman, Allan M.;
Duong, Tina;
De Sanctis, Roberto;
Mazzone, Elena Stacy;
Milev, Evelin;
Rohwer, Annemarie;
Civitello, Matthew;
Pane, Marika;
Antonaci, Laura;
Frongia, Anna Lia;
Sframeli, Maria;
Vita, Gian Luca

Publication type:

Article

Conference report on contractures in musculoskeletal and neurological conditions.

Published in:

2020

By:

Nuckolls, Glen H.;
Kinnett, Kathi;
Dayanidhi, Sudarshan;
Domenighetti, Andrea A.;
Duong, Tina;
Hathout, Yetrib;
Lawlor, Michael W.;
Lee, Sabrina S. M.;
Magnusson, S. Peter;
McDonald, Craig M.;
McNally, Elizabeth M.;
Miller, Natalie F.;
Olwin, Bradley B.;
Raghavan, Preeti;
Roberts, Thomas J.;
Rutkove, Seward B.;
Sarwark, John F.;
Senesac, Claudia R.;
Vogel, Leslie F.;
Walter, Glenn A.

Publication type:

journal article

Revised upper limb module for spinal muscular atrophy: 12 month changes.

Published in:

2019

By:

Pera, Maria Carmela;
Coratti, Giorgia;
Mazzone, Elena S.;
Montes, Jacqueline;
Scoto, Mariacristina;
De Sanctis, Roberto;
Main, Marion;
Mayhew, Anna;
Muni Lofra, Robert;
Dunaway Young, Sally;
Glanzman, Allan M.;
Duong, Tina;
Pasternak, Amy;
Ramsey, Danielle;
Darras, Basil;
Day, John W.;
Finkel, Richard S.;
De Vivo, Darryl C.;
Sormani, Maria Pia;
Bovis, Francesca

Publication type:

journal article

Electrical impedance myography in individuals with collagen 6 and laminin α-2 congenital muscular dystrophy: a cross-sectional and 2-year analysis.

Published in:

2018

By:

Nichols, Carmel;
Jain, Minal S.;
Meilleur, Katherine G.;
Wu, Tianxia;
Collins, James;
Waite, Melissa R.;
Dastgir, Jahannaz;
Salman, Anam;
Donkervoort, Sandra;
Duong, Tina;
Keller, Katherine;
Leach, Meganne E.;
Lott, Donovan J.;
McGuire, Michelle N.;
Nelson, Leslie;
Rutkowski, Anne;
Vuillerot, Carole;
Bönnemann, Carsten G.;
Lehky, Tanya J.

Publication type:

journal article

Revised upper limb module for spinal muscular atrophy: Development of a new module.

Published in:

2017

By:

Mazzone, Elena S.;
Mayhew, Anna;
Montes, Jacqueline;
Ramsey, Danielle;
Fanelli, Lavinia;
Young, Sally Dunaway;
Salazar, Rachel;
De Sanctis, Roberto;
Pasternak, Amy;
Glanzman, Allan;
Coratti, Giorgia;
Civitello, Matthew;
Forcina, Nicola;
Gee, Richard;
Duong, Tina;
Pane, Marika;
Scoto, Mariacristina;
Pera, Maria Carmela;
Messina, Sonia;
Tennekoon, Gihan

Publication type:

journal article

JEWELFISH: 24-month results from an open-label study in non-treatment-naïve patients with SMA receiving treatment with risdiplam.

Published in:

Journal of Neurology, 2024, v. 271, n. 8, p. 4871, doi. 10.1007/s00415-024-12318-z

By:

Chiriboga, Claudia A.;
Bruno, Claudio;
Duong, Tina;
Fischer, Dirk;
Mercuri, Eugenio;
Kirschner, Janbernd;
Kostera-Pruszczyk, Anna;
Jaber, Birgit;
Gorni, Ksenija;
Kletzl, Heidemarie;
Carruthers, Imogen;
Martin, Carmen;
Scalco, Renata S.;
Fontoura, Paulo;
Muntoni, Francesco

Publication type:

Article

Towards regulatory endorsement of drug development tools to promote the application of model-informed drug development in Duchenne muscular dystrophy.

Published in:

Journal of Pharmacokinetics & Pharmacodynamics, 2019, v. 46, n. 5, p. 441, doi. 10.1007/s10928-019-09642-7

By:

Conrado, Daniela J.;
Larkindale, Jane;
Berg, Alexander;
Hill, Micki;
Burton, Jackson;
Abrams, Keith R.;
Abresch, Richard T.;
Bronson, Abby;
Chapman, Douglass;
Crowther, Michael;
Duong, Tina;
Gordish-Dressman, Heather;
Harnisch, Lutz;
Henricson, Erik;
Kim, Sarah;
McDonald, Craig M.;
Schmidt, Stephan;
Vong, Camille;
Wang, Xiaoxing;
Wong, Brenda L.

Publication type:

Article

Consensus Guidelines for Improving Quality of Assessment and Training for Neuromuscular Diseases.

Published in:

Frontiers in Genetics, 2021, v. 12, p. 1, doi. 10.3389/fgene.2021.735936

By:

Duong, Tina;
Krosschell, Kristin J.;
James, Meredith K.;
Nelson, Leslie;
Alfano, Lindsay N.;
Eichinger, Katy;
Mazzone, Elena;
Rose, Kristy;
Lowes, Linda P.;
Mayhew, Anna;
Florence, Julaine;
King, Wendy;
Senesac, Claudia R.;
Eagle, Michelle

Publication type:

Article

Long-term efficacy, safety, and patient-reported outcomes of apitegromab in patients with spinal muscular atrophy: results from the 36-month TOPAZ study.

Published in:

Frontiers in Neurology, 2024, p. 1, doi. 10.3389/fneur.2024.1419791

By:

Crawford, Thomas O.;
Day, John W.;
De Vivo, Darryl C.;
Krueger, Jena M.;
Mercuri, Eugenio;
Nascimento, Andres;
Pasternak, Amy;
Mazzone, Elena Stacy;
Duong, Tina;
Guochen Song;
Marantz, Jing L.;
Baver, Scott;
Dongzi Yu;
Lan Liu;
Darras, Basil T.

Publication type:

Article

Long‐term natural history in type II and III spinal muscular atrophy: a 4‐year international study on the Hammersmith Functional Motor Scale Expanded.

Published in:

European Journal of Neurology, 2024, v. 31, n. 12, p. 1, doi. 10.1111/ene.16517

By:

Coratti, Giorgia;
Bovis, Francesca;
Pera, Maria Carmela;
Civitello, Matthew;
Rohwer, Annemarie;
Salmin, Francesca;
Glanzman, Allan M.;
Montes, Jacqueline;
Pasternak, Amy;
De Sanctis, Roberto;
Dunaway Young, Sally;
Duong, Tina;
Mizzoni, Irene;
Milev, Evelin;
Sframeli, Maria;
Morando, Simone;
Albamonte, Emilio;
D'Amico, Adele;
Catteruccia, Michela;
Brolatti, Noemi

Publication type:

Article

Determining minimal clinically important differences in the Hammersmith Functional Motor Scale Expanded for untreated spinal muscular atrophy patients: An international study.

Published in:

European Journal of Neurology, 2024, v. 31, n. 8, p. 1, doi. 10.1111/ene.16309

By:

Coratti, Giorgia;
Bovis, Francesca;
Pera, Maria Carmela;
Scoto, Mariacristina;
Montes, Jacqueline;
Pasternak, Amy;
Mayhew, Anna;
Muni‐Lofra, Robert;
Duong, Tina;
Rohwer, Annemarie;
Dunaway Young, Sally;
Civitello, Matthew;
Salmin, Francesca;
Mizzoni, Irene;
Morando, Simone;
Pane, Marika;
Albamonte, Emilio;
D'Amico, Adele;
Brolatti, Noemi;
Sframeli, Maria

Publication type:

Article

Serial casting for contractures in SMA: consensus derived guidelines for treatment.

Published in:

Frontiers in Neurology, 2025, p. 1, doi. 10.3389/fneur.2025.1502495

By:

Brown, Laurey;
Hoffman, Katie;
Corbo-Galli, Chiara;
Kelley, Carolyn;
Carry, Terri;
Civitello, Matt;
Coratti, Giorgia;
DeSanctis, Roberto;
Duong, Tina;
Driscoll, Brigid;
Flickinger, Jean;
Glanzman, Allan M.;
Jones, Jennifer;
Maczek, Elizabeth;
Moat, Dionne;
Montes, Jacqueline;
Muni-Lofra, Robert;
Nelson, Leslie;
Pasternak, Amy;
Valle, Melanie

Publication type:

Article

In Vitro Cytotoxicity of Antiresorptive and Antiangiogenic Compounds on Oral Tissues Contributing to MRONJ: Systematic Review.

Published in:

Biomolecules (2218-273X), 2023, v. 13, n. 6, p. 973, doi. 10.3390/biom13060973

By:

Guirguis, Robert H.;
Tan, Leonard P.;
Hicks, Rebecca M.;
Hasan, Aniqa;
Duong, Tina D.;
Hu, Xia;
Hng, Jordan Y. S.;
Hadi, Mohammad H.;
Owuama, Henry C.;
Matthyssen, Tamara;
McCullough, Michael;
Canfora, Federica;
Paolini, Rita;
Celentano, Antonio

Publication type:

Article

Ambulatory function in spinal muscular atrophy: Age-related patterns of progression.

Published in:

PLoS ONE, 2018, v. 13, n. 6, p. 1, doi. 10.1371/journal.pone.0199657

By:

Montes, Jacqueline;
McDermott, Michael P.;
Mirek, Elizabeth;
Mazzone, Elena S.;
Main, Marion;
Glanzman, Allan M.;
Duong, Tina;
Young, Sally Dunaway;
Salazar, Rachel;
Pasternak, Amy;
Gee, Richard;
De Sanctis, Roberto;
Coratti, Giorgia;
Forcina, Nicola;
Fanelli, Lavinia;
Ramsey, Danielle;
Milev, Evelin;
Civitello, Matthew;
Pane, Marika;
Pera, Maria Carmela

Publication type:

Article

Disease Trajectories in the Revised Hammersmith Scale in a Cohort of Untreated Patients with Spinal Muscular Atrophy types 2 and 3.

Published in:

Journal of Neuromuscular Diseases, 2024, v. 11, n. 3, p. 665, doi. 10.3233/JND-230211

By:

Wolfe, Amy;
Stimpson, Georgia;
Ramsey, Danielle;
Coratti, Giorgia;
Dunaway Young, Sally;
Mayhew, Anna;
Pane, Marika;
Rohwer, Annemarie;
Muni Lofra, Robert;
Duong, Tina;
O'Reilly, Emer;
Milev, Evelin;
Civitello, Matthew;
Sansone, Valeria A.;
D'Amico, Adele;
Bertini, Enrico;
Messina, Sonia;
Bruno, Claudio;
Albamonte, Emilio;
Mazzone, Elena

Publication type:

Article

Development of an International SMA Bulbar Assessment for Inter-professional Administration.

Published in:

Journal of Neuromuscular Diseases, 2023, v. 10, n. 4, p. 639, doi. 10.3233/JND-221672

By:

Dunaway Young, Sally;
McGrattan, Katlyn;
Johnson, Emily;
van der Heul, Marise;
Duong, Tina;
Bakke, Merete;
Werlauff, Ulla;
Pasternak, Amy;
Cattaneo, Camilla;
Hoffman, Katie;
Fanelli, Lavinia;
Breaks, Anne;
Allison, Kristen;
Baranello, Giovanni;
Finkel, Richard;
Coratti, Giorgia;
Lofra, Robert Muni

Publication type:

Article

Assessing Bulbar Function in Spinal Muscular Atrophy Using Patient-Reported Outcomes.

Published in:

Journal of Neuromuscular Diseases, 2023, v. 10, n. 2, p. 199, doi. 10.3233/JND-221573

By:

Dunaway Young, Sally;
Pasternak, Amy;
Duong, Tina;
McGrattan, Katlyn E.;
Stranberg, Sarah;
Maczek, Elizabeth;
Dias, Courtney;
Tang, Whitney;
Parker, Dana;
Levine, Alexis;
Rohan, Alyssa;
Wolford, Connie;
Martens, William;
McDermott, Michael P.;
Darras, Basil T.;
Day, John W.

Publication type:

Article

Motor Responses in Pediatric Pompe Disease in the ADVANCE Participant Cohort.

Published in:

Journal of Neuromuscular Diseases, 2022, v. 9, n. 6, p. 713, doi. 10.3233/JND-210784

By:

Duong, Tina;
Kishnani, Priya S.;
An Haack, Kristina;
Foster, Meredith C.;
Gibson, James B.;
Wilson, Catherine;
Hahn, Si Houn;
Hillman, Richard;
Kronn, David;
Leslie, Nancy D.;
Peña, Loren D.M.;
Sparks, Susan E.;
Stockton, David W.;
Tanpaiboon, Pranoot;
Day, John W.

Publication type:

Article

INCEPTUS Natural History, Run-in Study for Gene Replacement Clinical Trial in X-Linked Myotubular Myopathy.

Published in:

Journal of Neuromuscular Diseases, 2022, v. 9, n. 4, p. 503, doi. 10.3233/JND-210781

By:

Dowling, James J.;
Müller-Felber, Wolfgang;
Smith, Barbara K.;
Bönnemann, Carsten G.;
Kuntz, Nancy L.;
Muntoni, Francesco;
Servais, Laurent;
Alfano, Lindsay N.;
Beggs, Alan H.;
Bilder, Deborah A.;
Blaschek, Astrid;
Duong, Tina;
Graham, Robert J.;
Jain, Minal;
Lawlor, Michael W.;
Lee, Jun;
Coats, Julie;
Lilien, Charlotte;
Lowes, Linda P.;
MacBean, Victoria

Publication type:

Article

Knee Strength and Ankle Range of Motion Impacts on Timed Function Tests in Duchenne Muscular Dystrophy: In the Era of Glucocorticoids.

Published in:

Journal of Neuromuscular Diseases, 2022, v. 9, n. 1, p. 147, doi. 10.3233/JND-210724

By:

Duong, Tina;
Canbek, Jennifer;
Fernandez-Fernandez, Alicia;
Henricson, Erik;
Birkmeier, Marisa;
Siener, Catherine;
Tesi Rocha, Carolina;
McDonald, Craig;
Gordish-Dressman, Heather

Publication type:

Article

The Minimal Clinical Important Difference (MCID) in Annual Rate of Change of Timed Function Tests in Boys with DMD.

Published in:

Journal of Neuromuscular Diseases, 2021, v. 8, n. 6, p. 939, doi. 10.3233/JND-210646

By:

Duong, Tina;
Canbek, Jennifer;
Birkmeier, Marisa;
Nelson, Leslie;
Siener, Catherine;
Fernandez-Fernandez, Alicia;
Henricson, Erik;
McDonald, Craig M.;
Gordish-Dressman, Heather

Publication type:

Article

E-Health & Innovation to Overcome Barriers in Neuromuscular Diseases. Report from the 1st eNMD Congress: Nice, France, March 22-23, 2019.

Published in:

Journal of Neuromuscular Diseases, 2021, v. 8, n. 4, p. 743, doi. 10.3233/JND-210655

By:

Pini, Jonathan;
Siciliano, Gabriele;
Lahaut, Pauline;
Braun, Serge;
Segovia-Kueny, Sandrine;
Kole, Anna;
Hérnando, Ines;
Selb, Julij;
Schirinzi, Erika;
Duong, Tina;
Hogrel, Jean-Yves;
Serrano Olmedo, José Javier;
Vissing, John;
Servais, Laurent;
Yincent-Genod, Dominique;
Vuillerot, Carole;
Bannwarth, Sylvie;
Eggenspieler, Damien;
Vicart, Savine;
Diaz-Manera, Jordi

Publication type:

Article

Improving Care and Empowering Adults Living with SMA: A Call to Action in the New Treatment Era.

Published in:

Journal of Neuromuscular Diseases, 2021, v. 8, n. 4, p. 543, doi. 10.3233/JND-200611

By:

Walter, Maggie C.;
Chiriboga, Claudia A.;
Duong, Tina;
Goemans, Nathalie;
Mayhew, Anna;
Ouillade, Laëtitia;
Oskoui, Maryam;
Quinlivan, Ros;
Vázquez-Costa, Juan F.;
Vissing, John;
Servais, Laurent

Publication type:

Article

Assessing Dysferlinopathy Patients Over Three Years With a New Motor Scale.

Published in:

2021

By:

Jacobs, Marni B.;
James, Meredoith K.;
Lowes, Linda P.;
Alfano, Lindsay N.;
Eagle, Michelle;
Muni Lofra, Robert;
Moore, Ursula;
Feng, Jia;
Rufibach, Laura E.;
Rose, Kristy;
Duong, Tina;
Bello, Luca;
Pedrosa‐Hernández, Irene;
Holsten, Scott;
Sakamoto, Chikako;
Canal, Aurélie;
Sanchez‐Aguilera Práxedes, Nieves;
Thiele, Simone;
Siener, Catherine;
Vandevelde, Bruno

Publication type:

journal article

Clinical Variability in Spinal Muscular Atrophy Type III.

Published in:

Annals of Neurology, 2020, v. 88, n. 6, p. 1109, doi. 10.1002/ana.25900

By:

Coratti, Giorgia;
Messina, Sonia;
Lucibello, Simona;
Pera, Maria Carmela;
Montes, Jacqueline;
Pasternak, Amy;
Bovis, Francesca;
Exposito Escudero, Jessica;
Mazzone, Elena Stacy;
Mayhew, Anna;
Glanzman, Allan M.;
Young, Sally Dunaway;
Salazar, Rachel;
Duong, Tina;
Muni Lofra, Robert;
De Sanctis, Roberto;
Carnicella, Sara;
Milev, Evelin;
Civitello, Matthew;
Pane, Marika

Publication type:

Article

Genetic modifiers of ambulation in the cooperative international Neuromuscular research group Duchenne natural history study.

Published in:

Annals of Neurology, 2015, v. 77, n. 4, p. 684, doi. 10.1002/ana.24370

By:

Bello, Luca;
Kesari, Akanchha;
Gordish‐Dressman, Heather;
Cnaan, Avital;
Morgenroth, Lauren P.;
Punetha, Jaya;
Duong, Tina;
Henricson, Erik K.;
Pegoraro, Elena;
McDonald, Craig M.;
Hoffman, Eric P.

Publication type:

Article

Development of the Performance of the Upper Limb module for Duchenne muscular dystrophy.

Published in:

Developmental Medicine & Child Neurology, 2013, v. 55, n. 11, p. 1038, doi. 10.1111/dmcn.12213

By:

Mayhew, Anna;
Mazzone, Elena S;
Eagle, Michelle;
Duong, Tina;
Ash, Maria;
Decostre, Valerie;
Vandenhauwe, Marlene;
Klingels, Katrijn;
Florence, Julaine;
Main, Marion;
Bianco, Flaviana;
Henrikson, Erik;
Servais, Laurent;
Campion, Giles;
Vroom, Elizabeth;
Ricotti, Valeria;
Goemans, Natalie;
Mcdonald, Craig;
Mercuri, Eugenio

Publication type:

Article

Correction to: Understanding the relationship between the 32-item motor function measure and daily activities from an individual with spinal muscular atrophy and their caregivers' perspective: a two-part study.

Published in:

2021

By:

Duong, Tina;
Braid, Jessica;
Staunton, Hannah;
Barriere, Aurelie;
Petridis, Fani;
Reithinger, Johannes;
Cruz, Rosangel;
Jarecki, Jill;
De Lemus, Mencia;
Gusset, Nicole;
Broekgaarden, Ria;
Randhawa, Sharan;
Flynn, Jessica;
Arbuckle, Rob;
Reif, Sonia;
Yang, Lida;
De Martini, Angela;
Vuillerot, Carole

Publication type:

corrected article

Understanding the relationship between the 32-item motor function measure and daily activities from an individual with spinal muscular atrophy and their caregivers' perspective: a two-part study.

Published in:

BMC Neurology, 2021, v. 21, n. 1, p. 1, doi. 10.1186/s12883-021-02166-z

By:

Duong, Tina;
Braid, Jessica;
Staunton, Hannah;
Barriere, Aurelie;
Petridis, Fani;
Reithinger, Johannes;
Cruz, Rosangel;
Jarecki, Jill;
De Lemus, Mencia;
Gusset, Nicole;
Broekgaarden, Ria;
Randhawa, Sharan;
Flynn, Jessica;
Arbuckle, Rob;
Reif, Sonia;
Yang, Lida;
De Martini, Angela;
Vuillerot, Carole

Publication type:

Article

Understanding the relationship between the 32-item motor function measure and daily activities from an individual with spinal muscular atrophy and their caregivers' perspective: a two-part study.

Published in:

BMC Neurology, 2021, v. 21, n. 1, p. 1, doi. 10.1186/s12883-021-02166-z

By:

Duong, Tina;
Braid, Jessica;
Staunton, Hannah;
Barriere, Aurelie;
Petridis, Fani;
Reithinger, Johannes;
Cruz, Rosangel;
Jarecki, Jill;
De Lemus, Mencia;
Gusset, Nicole;
Broekgaarden, Ria;
Randhawa, Sharan;
Flynn, Jessica;
Arbuckle, Rob;
Reif, Sonia;
Yang, Lida;
De Martini, Angela;
Vuillerot, Carole

Publication type:

Article

Nusinersen in pediatric and adult patients with type III spinal muscular atrophy.

Published in:

Annals of Clinical & Translational Neurology, 2021, v. 8, n. 8, p. 1622, doi. 10.1002/acn3.51411

By:

Pera, Maria Carmela;
Coratti, Giorgia;
Bovis, Francesca;
Pane, Marika;
Pasternak, Amy;
Montes, Jacqueline;
Sansone, Valeria A.;
Dunaway Young, Sally;
Duong, Tina;
Messina, Sonia;
Mizzoni, Irene;
D'Amico, Adele;
Civitello, Matthew;
Glanzman, Allan M.;
Bruno, Claudio;
Salmin, Francesca;
Morando, Simone;
De Sanctis, Roberto;
Sframeli, Maria;
Antonaci, Laura

Publication type:

Article

Deletion of 12/15-Lipoxygenase Alters Macrophage and Islet Function in NOD-<i>Alox15<sup>null</sup></i> Mice, Leading to Protection against Type 1 Diabetes Development.

Published in:

PLoS ONE, 2013, v. 8, n. 2, p. 1, doi. 10.1371/journal.pone.0056763

By:

Green-Mitchell, Shamina M.;
Tersey, Sarah A.;
Cole, Banumathi K.;
Ma, Kaiwen;
Kuhn, Norine S.;
Cunningham, Tina Duong;
Maybee, Nelly A.;
Chakrabarti, Swarup K.;
McDuffie, Marcia;
Taylor-Fishwick, David A.;
Mirmira, Raghavendra G.;
Nadler, Jerry L.;
Morris, Margaret A.

Publication type:

Article

Correction to: Risdiplam in Patients Previously Treated with Other Therapies for Spinal Muscular Atrophy: An Interim Analysis from the JEWELFISH Study.

Published in:

Neurology & Therapy, 2023, v. 12, n. 5, p. 1799, doi. 10.1007/s40120-023-00503-7

By:

Chiriboga, Claudia A.;
Bruno, Claudio;
Duong, Tina;
Fischer, Dirk;
Mercuri, Eugenio;
Kirschner, Janbernd;
Kostera-Pruszczyk, Anna;
Jaber, Birgit;
Gorni, Ksenija;
Kletzl, Heidemarie;
Carruthers, Imogen;
Martin, Carmen;
Warren, Francis;
Scalco, Renata S.;
Wagner, Kathryn R.;
Muntoni, Francesco;
the JEWELFISH Study Group;
Deconinck, Nicolas;
Balikova, Irina;
Joniau, Inge

Publication type:

Article

Risdiplam in Patients Previously Treated with Other Therapies for Spinal Muscular Atrophy: An Interim Analysis from the JEWELFISH Study.

Published in:

Neurology & Therapy, 2023, v. 12, n. 2, p. 543, doi. 10.1007/s40120-023-00444-1

By:

Chiriboga, Claudia A.;
Bruno, Claudio;
Duong, Tina;
Fischer, Dirk;
Mercuri, Eugenio;
Kirschner, Janbernd;
Kostera-Pruszczyk, Anna;
Jaber, Birgit;
Gorni, Ksenija;
Kletzl, Heidemarie;
Carruthers, Imogen;
Martin, Carmen;
Warren, Francis;
Scalco, Renata S.;
Wagner, Kathryn R.;
Muntoni, Francesco

Publication type:

Article

The cooperative international neuromuscular research group Duchenne natural history study--a longitudinal investigation in the era of glucocorticoid therapy: design of protocol and the methods used.

Published in:

2013

By:

McDonald, Craig M;
Henricson, Erik K;
Abresch, R Ted;
Han, Jay J;
Escolar, Diana M;
Florence, Julaine M;
Duong, Tina;
Arrieta, Adrienne;
Clemens, Paula R;
Hoffman, Eric P;
Cnaan, Avital;
Cinrg Investigators

Publication type:

journal article

The cooperative international neuromuscular research group duchenne natural history study-a longitudinal investigation in the era of glucocorticoid therapy: Design of protocol and the methods used.

Published in:

Muscle & Nerve, 2013, v. 48, n. 1, p. 32, doi. 10.1002/mus.23807

By:

McDonald, Craig M.;
Henricson, Erik K.;
Abresch, R. Ted;
Han, Jay J.;
Escolar, Diana M.;
Florence, Julaine M.;
Duong, Tina;
Arrieta, Adrienne;
Clemens, Paula R.;
Hoffman, Eric P.;
Cnaan, Avital

Publication type:

Article

The cooperative international neuromuscular research group Duchenne natural history study: Glucocorticoid treatment preserves clinically meaningful functional milestones and reduces rate of disease progression as measured by manual muscle testing and other commonly used clinical trial outcome measures

Published in:

Muscle & Nerve, 2013, v. 48, n. 1, p. 55, doi. 10.1002/mus.23808

By:

Henricson, Erik K.;
Abresch, R. Ted;
Cnaan, Avital;
Hu, Fengming;
Duong, Tina;
Arrieta, Adrienne;
Han, Jay;
Escolar, Diana M.;
Florence, Julaine M.;
Clemens, Paula R.;
Hoffman, Eric P.;
McDonald, Craig M.

Publication type:

Article

Content validity and clinical meaningfulness of the HFMSE in spinal muscular atrophy.

Published in:

2017

By:

Pera, Maria C.;
Coratti^, Giorgia;
Forcina, Nicola;
Mazzone, Elena S.;
Scoto, Mariacristina;
Montes, Jacqueline;
Pasternak, Amy;
Mayhew, Anna;
Messina, Sonia;
Sframeli, Maria;
Main, Marion;
Lofra, Robert Muni;
Duong, Tina;
Ramsey, Danielle;
Dunaway, Sally;
Salazar, Rachel;
Fanelli^, Lavinia;
Civitello, Matthew;
de Sanctis, Roberto;
Antonaci, Laura

Publication type:

journal article