Works matching IS 22143599 AND DT 2023 AND VI 10 AND IP 6

Results: 15

Reply to F. Muntoni et al.: "In response to P.R. Clemens et al., Efficacy and Safety of Viltolarsen in Boys with Duchenne Muscular Dystrophy: Results From the Phase 2, Open-Label, 4-Year Extension Study, and Long-Term Functional Efficacy and Safety of Viltolarsen in Patients with Duchenne Muscular Dystrophy

Published in:

2023

By:

Clemens, Paula R.;
Hoffman, Eric P.

Publication type:

Letter

Endocrine and Bone Monitoring in Boys with Duchenne Muscular Dystrophy; Do we adhere to the standards of care?

Published in:

Journal of Neuromuscular Diseases, 2023, v. 10, n. 6, p. 1143, doi. 10.3233/JND-230144

By:

Henderson, A.;
Harley, G.;
Horrocks, I.;
Joseph, S.;
Dunne, J.;
Pysden, K.;
Mushtaq, T.;
Wong, S.C.;
Childs, A.M.

Publication type:

Article

Letter to the Editor: In response to P.R. Clemens et al., Efficacy and Safety of Viltolarsen in Boys with Duchenne Muscular Dystrophy: Results From the Phase 2, Open-Label, 4-Year Extension Study, and Long-Term Functional Efficacy and Safety of Viltolarsen in Patients with Duchenne Muscular Dystrophy

Published in:

2023

By:

Muntoni, Francesco;
Straub, Volker;
Servais, Laurent;
Mercuri, Eugenio

Publication type:

Letter

Considering the Promise of Vamorolone for Treating Duchenne Muscular Dystrophy.

Published in:

Journal of Neuromuscular Diseases, 2023, v. 10, n. 6, p. 1013, doi. 10.3233/JND-230161

By:

Grounds, Miranda D.;
Lloyd, Erin M.

Publication type:

Article

Characterising the Genetic Landscape of Amyotrophic Lateral Sclerosis: A Catalogue and Assessment of Over 1,000 Published Genetic Variants.

Published in:

Journal of Neuromuscular Diseases, 2023, v. 10, n. 6, p. 1127, doi. 10.3233/JND-230148

By:

McCann, Emily P.;
Grima, Natalie;
Fifita, Jennifer A.;
Chan Moi Fat, Sandrine;
Lehnert, Klaus;
Henden, Lyndal;
Blair, Ian P.;
Williams, Kelly L.

Publication type:

Article

Humanization of the mdx Mouse Phenotype for Duchenne Muscular Dystrophy Modeling: A Metabolic Perspective.

Published in:

Journal of Neuromuscular Diseases, 2023, v. 10, n. 6, p. 1003, doi. 10.3233/JND-230126

By:

Donen, Graham;
Milad, Nadia;
Bernatchez, Pascal

Publication type:

Article

Resistance Exercise Training Rescues Mitochondrial Dysfunction in Skeletal Muscle of Patients with Myotonic Dystrophy Type 1.

Published in:

Journal of Neuromuscular Diseases, 2023, v. 10, n. 6, p. 1111, doi. 10.3233/JND-230099

By:

Di Leo, Valeria;
Lawless, Conor;
Roussel, Marie-Pier;
Gomes, Tiago B.;
Gorman, Gráinne S.;
Russell, Oliver M.;
Tuppen, Helen A.L.;
Duchesne, Elise;
Vincent, Amy E.

Publication type:

Article

A Mixed-method Approach to Develop an Ambulatory Module of the SMA Independence Scale.

Published in:

Journal of Neuromuscular Diseases, 2023, v. 10, n. 6, p. 1093, doi. 10.3233/JND-230096

By:

Staunton, Hannah;
Cleanthous, Sophie;
Teodoro, Vanda;
Barrett, Louise;
Braid, Jessica;
Ewens, Bethany;
Cano, Stefan;
Baranello, Giovanni;
Kirschner, Janbernd;
Belter, Lisa;
Mayhew, Anna

Publication type:

Article

Longitudinal Changes in Neuropsychological Functioning in Japanese Patients with Myotonic Dystrophy Type 1: A Five Year Follow-Up Study.

Published in:

Journal of Neuromuscular Diseases, 2023, v. 10, n. 6, p. 1083, doi. 10.3233/JND-230083

By:

Fujino, Haruo;
Suwazono, Shugo;
Ueda, Yukihiko;
Kobayashi, Michio;
Nakayama, Takahiro;
Imura, Osamu;
Matsumura, Tsuyoshi;
Takahashi, Masanori P.

Publication type:

Article

Respiratory Insufficiency in Neuromuscular Disease (RIND): A Delphi Study to Establish Consensus Criteria to Define and Diagnose Hypoventilation in Pediatric Neuromuscular Disease.

Published in:

Journal of Neuromuscular Diseases, 2023, v. 10, n. 6, p. 1075, doi. 10.3233/JND-230053

By:

Mayer, Oscar Henry;
Amin, Reshma;
Sawnani, Hemant;
Shell, Richard;
Katz, Sherri Lynne

Publication type:

Article

Oral Dantrolene Reduces Myalgia and Hyperckemia in a Child with RYR1-Related Exertional Myalgia/Rhabdomyolysis.

Published in:

Journal of Neuromuscular Diseases, 2023, v. 10, n. 6, p. 1145, doi. 10.3233/JND-230007

By:

de Lima Silva, Elis Vanessa;
Donis, Karina Carvalho;
Machado, Fabiana Rita Camara;
Simão Medeiros, Leonardo;
Aschoff, Carlos Alberto de Moura;
de Souza, Carolina Fischinger Moura;
Poswar, Fabiano de Oliveira;
Saute, Jonas Alex Morales

Publication type:

Article

SELENON-Related Myopathy Across the Life Span, a Cross-Sectional Study for Preparing Trial Readiness.

Published in:

Journal of Neuromuscular Diseases, 2023, v. 10, n. 6, p. 1055, doi. 10.3233/JND-221673

By:

Bouman, Karlijn;
Groothuis, Jan T.;
Doorduin, Jonne;
van Alfen, Nens;
Udink ten Cate, Floris E.A.;
van den Heuvel, Frederik M.A.;
Nijveldt, Robin;
Kamsteeg, Erik-Jan;
Dittrich, Anne T.M.;
Draaisma, Jos M.T.;
Janssen, Mirian C.H.;
van Engelen, Baziel G.M.;
Erasmus, Corrie E.;
Voermans, Nicol C.

Publication type:

Article

Biomarkers in Duchenne Muscular Dystrophy: Current Status and Future Directions.

Published in:

Journal of Neuromuscular Diseases, 2023, v. 10, n. 6, p. 987, doi. 10.3233/JND-221666

By:

Fortunato, Fernanda;
Ferlini, Alessandra

Publication type:

Article

Towards the Identification of Biomarkers for Muscle Function Improvement in Myotonic Dystrophy Type 1.

Published in:

Journal of Neuromuscular Diseases, 2023, v. 10, n. 6, p. 1041, doi. 10.3233/JND-221645

By:

Aoussim, Amira;
Légaré, Cécilia;
Roussel, Marie-Pier;
Madore, Anne-Marie;
Morissette, Mathieu C.;
Laprise, Catherine;
Duchesne, Elise

Publication type:

Article

A Targeted Approach for Evaluating DUX4-Regulated Proteins as Potential Serum Biomarkers for Facioscapulohumeral Muscular Dystrophy Using Immunoassay Proteomics.

Published in:

Journal of Neuromuscular Diseases, 2023, v. 10, n. 6, p. 1031, doi. 10.3233/JND-221636

By:

Campbell, Amy E.;
Arjomand, Jamshid;
King, Oliver D.;
Tawil, Rabi;
Jagannathan, Sujatha

Publication type:

Article