Works matching IS 2214-3599 AND VI 12 AND IP 3 AND DT 2025

Results: 12

Preclinical development of genome editing to treat Duchenne muscular dystrophy by exon skipping.

Published in:

Journal of Neuromuscular Diseases, 2025, v. 12, n. 3, p. 424, doi. 10.1177/22143602251326993

By:

Padmaswari, Made Harumi;
Agrawal, Shilpi;
Nelson, Christopher E

Publication type:

Article

Patient-reported assessment of bulbar function in spinal muscular atrophy (SMA): Validation of a self-report scale.

Published in:

Journal of Neuromuscular Diseases, 2025, v. 12, n. 3, p. 394, doi. 10.1177/22143602251325741

By:

Lefton-Greif, Maureen A;
Belter, Lisa;
Jarecki, Jill;
Schroth, Mary;
Zeng, Yong;
Crawford, Thomas O;
Eaton, Cyd

Publication type:

Article

Factors affecting desired participation in transition to an adult life with Duchenne muscular dystrophy (DMD).

Published in:

Journal of Neuromuscular Diseases, 2025, v. 12, n. 3, p. 353, doi. 10.1177/22143602251324847

By:

Merkenhof, Laura JB;
Veenhuizen, Yvonne;
Vroom, Elizabeth;
Sterenberg, Greet;
Hesseling, Wendy CHM;
Groothuis, Jan T;
Cup, Edith H;
Houwen- van Opstal, Saskia LS

Publication type:

Article

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

Initiating non-invasive ventilation in patients with Amyotrophic Lateral Sclerosis in The Netherlands: A centralised approach to respiratory care.

Published in:

Journal of Neuromuscular Diseases, 2025, v. 12, n. 3, p. 372, doi. 10.1177/22143602251319167

By:

Jaspers Focks, Rineke JH;
Helleman, Jochem;
van den Berg, Leonard H;
Visser-Meily, Johanna MA;
Gaytant, Michael A;
Wijkstra, Peter J;
Beelen, Anita

Publication type:

Article

Recent progress in oculopharyngodistal myopathy research from clinical and genetic viewpoints.

Published in:

Journal of Neuromuscular Diseases, 2025, v. 12, n. 3, p. 303, doi. 10.1177/22143602251319164

By:

Ishiura, Hiroyuki

Publication type:

Article

Titinopathies: Phenotype – genotype heterogeneity in an Indian cohort.

Published in:

Journal of Neuromuscular Diseases, 2025, v. 12, n. 3, p. 364, doi. 10.1177/22143602241313119

By:

Baskar, Dipti;
Vengalil, Seena;
Polavarapu, Kiran;
Preethish-Kumar, Veeramani;
Nashi, Saraswati;
Arunachal, Gautham;
Srivastava, Kosha;
Desai, Vaishnavi;
Thomas, Priya Treesa;
Keerthipriya, Muddasu Suhasini;
Huddar, Akshata;
Unnikrishnan, Gopikrishnan;
Anjanappa, Ram Murthy;
Nalini, Atchayaram

Publication type:

Article

The participants' perspective on facioscapulohumeral muscular dystrophy trials in The Netherlands – A qualitative study.

Published in:

Journal of Neuromuscular Diseases, 2025, v. 12, n. 3, p. 382, doi. 10.1177/22143602241313117

By:

Stinissen, Lizan;
Kools, Joost;
Bouma, Sietse;
Lenssen, Emma;
Sanders, Eline;
Lanser, Anke;
de Haas, Ria;
van Engelen, Baziel GM;
Oortwijn, Wija;
Voermans, Nicol C

Publication type:

Article

Visualizing ambulatory performance by age and rates of decline among patients with Duchenne muscular dystrophy.

Published in:

Journal of Neuromuscular Diseases, 2025, v. 12, n. 3, p. 435, doi. 10.1177/22143602241313116

By:

Mayhew, Anna G;
Signorovitch, James;
Johnson, Michaela;
Frean, Molly;
Ward, Susan J;
Posner, Nate;
Merla, Valeria;
Mahn, Matthias;
Stimpson, Georgia;
Guglieri, Michela;
Straub, Volker;
Muni-Lofra, Robert;
Manzur, Adnan;
Baranello, Giovanni;
Muntoni, Francesco

Publication type:

Article

Physical training of wheelchair users with neuromuscular disorders: A systematic review.

Published in:

Journal of Neuromuscular Diseases, 2025, v. 12, n. 3, p. 330, doi. 10.1177/22143602241313114

By:

Poulsen, Nanna Scharff;
Kraglund, Lærke Rykær;
Vissing, John

Publication type:

Article

A state-of-the-art review of registries in spinal muscular atrophy: A valuable resource for clinical research.

Published in:

Journal of Neuromuscular Diseases, 2025, v. 12, n. 3, p. 312, doi. 10.1177/22143602241313113

By:

Balaji, Lakshmi;
Farrar, Michelle A;
Yiu, Eppie M;
Kariyawasam, Didu

Publication type:

Article

Comprehensive multidisciplinary care for adult Duchenne muscular dystrophy in South Korea.

Published in:

Journal of Neuromuscular Diseases, 2025, v. 12, n. 3, p. 342, doi. 10.1177/22143602241304996

By:

Choi, Won Ah;
Park, Hee Jae;
Kang, Seong-Woong;
Cho, Sung-Rae;
Cho, Han Eol

Publication type:

Article