Detalhe da pesquisa
1.
Functional abilities, respiratory and cardiac function in a large cohort of adults with Duchenne muscular dystrophy treated with glucocorticoids.
Eur J Neurol
; 31(6): e16267, 2024 Jun.
Artigo
em Inglês
| MEDLINE | ID: mdl-38556893
2.
Comparison of strength testing modalities in dysferlinopathy.
Muscle Nerve
; 66(2): 159-166, 2022 08.
Artigo
em Inglês
| MEDLINE | ID: mdl-35506767
3.
Cardiac and pulmonary findings in dysferlinopathy: A 3-year, longitudinal study.
Muscle Nerve
; 65(5): 531-540, 2022 05.
Artigo
em Inglês
| MEDLINE | ID: mdl-35179231
4.
Muscle MRI in patients with dysferlinopathy: pattern recognition and implications for clinical trials.
J Neurol Neurosurg Psychiatry
; 89(10): 1071-1081, 2018 10.
Artigo
em Inglês
| MEDLINE | ID: mdl-29735511
5.
Activity rating scales in adult muscle disease: what do they actually measure?
Muscle Nerve
; 50(1): 14-23, 2014 Jul.
Artigo
em Inglês
| MEDLINE | ID: mdl-24122732
6.
Activity rating scales in adult muscle disease: how well do they actually measure?
Muscle Nerve
; 50(1): 24-33, 2014 Jul.
Artigo
em Inglês
| MEDLINE | ID: mdl-24123139
7.
Teenage exercise is associated with earlier symptom onset in dysferlinopathy: a retrospective cohort study.
J Neurol Neurosurg Psychiatry
; 89(11): 1224-1226, 2018 11.
Artigo
em Inglês
| MEDLINE | ID: mdl-29378789
8.
Longitudinal Analysis of Respiratory Function of Different Types of Limb Girdle Muscular Dystrophies Reveals Independent Trajectories.
Neurol Genet
; 9(4): e200084, 2023 Aug.
Artigo
em Inglês
| MEDLINE | ID: mdl-37440793
9.
Myostatin and follistatin as monitoring and prognostic biomarkers in dysferlinopathy.
Neuromuscul Disord
; 33(2): 199-207, 2023 02.
Artigo
em Inglês
| MEDLINE | ID: mdl-36689846
10.
Development of a standard of care for patients with valosin-containing protein associated multisystem proteinopathy.
Orphanet J Rare Dis
; 17(1): 23, 2022 01 29.
Artigo
em Inglês
| MEDLINE | ID: mdl-35093159
11.
Validation of the North Star Assessment for Limb-Girdle Type Muscular Dystrophies.
Phys Ther
; 102(10)2022 10 06.
Artigo
em Inglês
| MEDLINE | ID: mdl-35932452
12.
Three-year quantitative magnetic resonance imaging and phosphorus magnetic resonance spectroscopy study in lower limb muscle in dysferlinopathy.
J Cachexia Sarcopenia Muscle
; 13(3): 1850-1863, 2022 06.
Artigo
em Inglês
| MEDLINE | ID: mdl-35373496
13.
Efficacy and Safety of Vamorolone vs Placebo and Prednisone Among Boys With Duchenne Muscular Dystrophy: A Randomized Clinical Trial.
JAMA Neurol
; 79(10): 1005-1014, 2022 10 01.
Artigo
em Inglês
| MEDLINE | ID: mdl-36036925
14.
Assessing the Relationship of Patient Reported Outcome Measures With Functional Status in Dysferlinopathy: A Rasch Analysis Approach.
Front Neurol
; 13: 828525, 2022.
Artigo
em Inglês
| MEDLINE | ID: mdl-35359643
15.
Functional outcome measures in young, steroid-naïve boys with Duchenne muscular dystrophy.
Neuromuscul Disord
; 32(6): 460-467, 2022 06.
Artigo
em Inglês
| MEDLINE | ID: mdl-35618576
16.
Remote Delivery of Motor Function Assessment and Training for Clinical Trials in Neuromuscular Disease: A Response to the COVID-19 Global Pandemic.
Front Genet
; 12: 735538, 2021.
Artigo
em Inglês
| MEDLINE | ID: mdl-34790223
17.
Consensus Guidelines for Improving Quality of Assessment and Training for Neuromuscular Diseases.
Front Genet
; 12: 735936, 2021.
Artigo
em Inglês
| MEDLINE | ID: mdl-34858470
18.
Miyoshi myopathy and limb girdle muscular dystrophy R2 are the same disease.
Neuromuscul Disord
; 31(4): 265-280, 2021 04.
Artigo
em Inglês
| MEDLINE | ID: mdl-33610434
19.
Natural history of limb girdle muscular dystrophy R9 over 6 years: searching for trial endpoints.
Ann Clin Transl Neurol
; 6(6): 1033-1045, 2019 Jun.
Artigo
em Inglês
| MEDLINE | ID: mdl-31211167
20.
Assessment of disease progression in dysferlinopathy: A 1-year cohort study.
Neurology
; 2019 Jan 09.
Artigo
em Inglês
| MEDLINE | ID: mdl-30626655