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.
Repeated intravenous cardiosphere-derived cell therapy in late-stage Duchenne muscular dystrophy (HOPE-2): a multicentre, randomised, double-blind, placebo-controlled, phase 2 trial.
Lancet
; 399(10329): 1049-1058, 2022 03 12.
Artigo
em Inglês
| MEDLINE | ID: mdl-35279258
3.
Assessing Dysferlinopathy Patients Over Three Years With a New Motor Scale.
Ann Neurol
; 89(5): 967-978, 2021 05.
Artigo
em Inglês
| MEDLINE | ID: mdl-33576057
4.
Effect of Different Corticosteroid Dosing Regimens on Clinical Outcomes in Boys With Duchenne Muscular Dystrophy: A Randomized Clinical Trial.
JAMA
; 327(15): 1456-1468, 2022 04 19.
Artigo
em Inglês
| MEDLINE | ID: mdl-35381069
5.
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
6.
Prophylactic oral bisphosphonate therapy in duchenne muscular dystrophy.
Muscle Nerve
; 54(1): 79-85, 2016 06.
Artigo
em Inglês
| MEDLINE | ID: mdl-26599341
7.
Two recurrent mutations are associated with GNE myopathy in the North of Britain.
J Neurol Neurosurg Psychiatry
; 85(12): 1359-65, 2014 Dec.
Artigo
em Inglês
| MEDLINE | ID: mdl-24695763
8.
Ataluren treatment of patients with nonsense mutation dystrophinopathy.
Muscle Nerve
; 50(4): 477-87, 2014 Oct.
Artigo
em Inglês
| MEDLINE | ID: mdl-25042182
9.
ANO5 gene analysis in a large cohort of patients with anoctaminopathy: confirmation of male prevalence and high occurrence of the common exon 5 gene mutation.
Hum Mutat
; 34(8): 1111-8, 2013 Aug.
Artigo
em Inglês
| MEDLINE | ID: mdl-23606453
10.
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
11.
Magnetic resonance imaging in Duchenne muscular dystrophy: longitudinal assessment of natural history over 18 months.
Muscle Nerve
; 48(4): 586-8, 2013 Oct.
Artigo
em Inglês
| MEDLINE | ID: mdl-23620230
12.
The 6-minute walk test and other clinical endpoints in duchenne muscular dystrophy: reliability, concurrent validity, and minimal clinically important differences from a multicenter study.
Muscle Nerve
; 48(3): 357-68, 2013 Sep.
Artigo
em Inglês
| MEDLINE | ID: mdl-23674289
13.
The 6-minute walk test and other endpoints in Duchenne muscular dystrophy: longitudinal natural history observations over 48 weeks from a multicenter study.
Muscle Nerve
; 48(3): 343-56, 2013 Sep.
Artigo
em Inglês
| MEDLINE | ID: mdl-23681930
14.
Detecting meaningful change using the North Star Ambulatory Assessment in Duchenne muscular dystrophy.
Dev Med Child Neurol
; 55(11): 1046-52, 2013 Nov.
Artigo
em Inglês
| MEDLINE | ID: mdl-23909763
15.
Development of the Performance of the Upper Limb module for Duchenne muscular dystrophy.
Dev Med Child Neurol
; 55(11): 1038-45, 2013 Nov.
Artigo
em Inglês
| MEDLINE | ID: mdl-23902233
16.
Mesenchymal lineage stem cells have pronounced anti-inflammatory effects in the twitcher mouse model of Krabbe's disease.
Stem Cells
; 29(1): 67-77, 2011 Jan.
Artigo
em Inglês
| MEDLINE | ID: mdl-21280158
17.
A founder mutation in Anoctamin 5 is a major cause of limb-girdle muscular dystrophy.
Brain
; 134(Pt 1): 171-182, 2011 Jan.
Artigo
em Inglês
| MEDLINE | ID: mdl-21186264
18.
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
19.
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
20.
Moving towards meaningful measurement: Rasch analysis of the North Star Ambulatory Assessment in Duchenne muscular dystrophy.
Dev Med Child Neurol
; 53(6): 535-42, 2011 Jun.
Artigo
em Inglês
| MEDLINE | ID: mdl-21410696