| nr |
titel |
auteur |
tijdschrift |
jaar |
jaarg. |
afl. |
pagina('s) |
type |
| 1 |
A Mixed-method Approach to Develop an Ambulatory Module of the SMA Independence Scale
|
Staunton, Hannah
|
|
|
10 |
6 |
p. 1093-1109
|
artikel
|
| 2 |
A Targeted Approach for Evaluating DUX4-Regulated Proteins as Potential Serum Biomarkers for Facioscapulohumeral Muscular Dystrophy Using Immunoassay Proteomics
|
Campbell, Amy E.
|
|
|
10 |
6 |
p. 1031-1040
|
artikel
|
| 3 |
Biomarkers in Duchenne Muscular Dystrophy: Current Status and Future Directions
|
Fortunato, Fernanda
|
|
|
10 |
6 |
p. 987-1002
|
artikel
|
| 4 |
Characterising the Genetic Landscape of Amyotrophic Lateral Sclerosis: A Catalogue and Assessment of Over 1,000 Published Genetic Variants
|
McCann, Emily P.
|
|
|
10 |
6 |
p. 1127-1141
|
artikel
|
| 5 |
Considering the Promise of Vamorolone for Treating Duchenne Muscular Dystrophy
|
Grounds, Miranda D.
|
|
|
10 |
6 |
p. 1013-1030
|
artikel
|
| 6 |
Endocrine and Bone Monitoring in Boys with Duchenne Muscular Dystrophy; Do we adhere to the standards of care?
|
Henderson, A.
|
|
|
10 |
6 |
p. 1143-1144
|
artikel
|
| 7 |
Humanization of the mdx Mouse Phenotype for Duchenne Muscular Dystrophy Modeling: A Metabolic Perspective
|
Donen, Graham
|
|
|
10 |
6 |
p. 1003-1012
|
artikel
|
| 8 |
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
|
Muntoni, Francesco
|
|
|
10 |
6 |
p. 1151-1153
|
artikel
|
| 9 |
Longitudinal Changes in Neuropsychological Functioning in Japanese Patients with Myotonic Dystrophy Type 1: A Five Year Follow-Up Study
|
Fujino, Haruo
|
|
|
10 |
6 |
p. 1083-1092
|
artikel
|
| 10 |
Oral Dantrolene Reduces Myalgia and Hyperckemia in a Child with RYR1-Related Exertional Myalgia/Rhabdomyolysis
|
de Lima Silva, Elis Vanessa
|
|
|
10 |
6 |
p. 1145-1149
|
artikel
|
| 11 |
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
|
Clemens, Paula R.
|
|
|
10 |
6 |
p. 1155-1157
|
artikel
|
| 12 |
Resistance Exercise Training Rescues Mitochondrial Dysfunction in Skeletal Muscle of Patients with Myotonic Dystrophy Type 1
|
Di Leo, Valeria
|
|
|
10 |
6 |
p. 1111-1126
|
artikel
|
| 13 |
Respiratory Insufficiency in Neuromuscular Disease (RIND): A Delphi Study to Establish Consensus Criteria to Define and Diagnose Hypoventilation in Pediatric Neuromuscular Disease
|
Mayer, Oscar Henry
|
|
|
10 |
6 |
p. 1075-1082
|
artikel
|
| 14 |
SELENON-Related Myopathy Across the Life Span, a Cross-Sectional Study for Preparing Trial Readiness
|
Bouman, Karlijn
|
|
|
10 |
6 |
p. 1055-1074
|
artikel
|
| 15 |
Towards the Identification of Biomarkers for Muscle Function Improvement in Myotonic Dystrophy Type 1
|
Aoussim, Amira
|
|
|
10 |
6 |
p. 1041-1053
|
artikel
|
Tijdschrift beschrijving