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
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
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
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