Detalhe da pesquisa
1.
Long-term safety and functional outcomes of delandistrogene moxeparvovec gene therapy in patients with Duchenne muscular dystrophy: A phase 1/2a nonrandomized trial.
Muscle Nerve
; 69(1): 93-98, 2024 Jan.
Artigo
em Inglês
| MEDLINE | ID: mdl-37577753
2.
Current status of clinical outcome measures in inclusion body myositis: a systematised review.
Clin Exp Rheumatol
; 41(2): 370-378, 2023 Mar.
Artigo
em Inglês
| MEDLINE | ID: mdl-36762744
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.
Genotype-phenotype correlations in valosin-containing protein disease: a retrospective muticentre study.
J Neurol Neurosurg Psychiatry
; 2022 07 27.
Artigo
em Inglês
| MEDLINE | ID: mdl-35896379
5.
Comparison of strength testing modalities in dysferlinopathy.
Muscle Nerve
; 66(2): 159-166, 2022 08.
Artigo
em Inglês
| MEDLINE | ID: mdl-35506767
6.
Measuring change in inclusion body myositis: clinical assessments versus imaging.
Clin Exp Rheumatol
; 40(2): 404-413, 2022 Feb.
Artigo
em Inglês
| MEDLINE | ID: mdl-35225227
7.
ACTIVE (Ability Captured Through Interactive Video Evaluation) workspace volume video game to quantify meaningful change in spinal muscular atrophy.
Dev Med Child Neurol
; 62(3): 303-309, 2020 03.
Artigo
em Inglês
| MEDLINE | ID: mdl-30963554
8.
Twice-weekly glucocorticosteroids in infants and young boys with Duchenne muscular dystrophy.
Muscle Nerve
; 59(6): 650-657, 2019 06.
Artigo
em Inglês
| MEDLINE | ID: mdl-30706490
9.
Paucity of bulbar function measures in inclusion body myositis trials. Reply to: Current status of clinical outcome measures in inclusion body myositis: a systematised review.
Clin Exp Rheumatol
; 41(2): 399, 2023 03.
Artigo
em Inglês
| MEDLINE | ID: mdl-36700648
10.
Follistatin Gene Therapy for Sporadic Inclusion Body Myositis Improves Functional Outcomes.
Mol Ther
; 25(4): 870-879, 2017 04 05.
Artigo
em Inglês
| MEDLINE | ID: mdl-28279643
11.
Longitudinal effect of eteplirsen versus historical control on ambulation in Duchenne muscular dystrophy.
Ann Neurol
; 79(2): 257-71, 2016 Feb.
Artigo
em Inglês
| MEDLINE | ID: mdl-26573217
12.
Modeling functional decline over time in sporadic inclusion body myositis.
Muscle Nerve
; 55(4): 526-531, 2017 04.
Artigo
em Inglês
| MEDLINE | ID: mdl-27511790
13.
Random forest: random results or meaningful insights for patients with facioscapulohumeral muscular dystrophy?
Brain
; 144(11): 3288-3290, 2021 12 16.
Artigo
em Inglês
| MEDLINE | ID: mdl-34636841
14.
Clinical trial readiness in non-ambulatory boys and men with duchenne muscular dystrophy: MDA-DMD network follow-up.
Muscle Nerve
; 54(4): 681-9, 2016 10.
Artigo
em Inglês
| MEDLINE | ID: mdl-26930423
15.
A phase 1/2a follistatin gene therapy trial for becker muscular dystrophy.
Mol Ther
; 23(1): 192-201, 2015 Jan.
Artigo
em Inglês
| MEDLINE | ID: mdl-25322757
16.
Reliability and validity of active-seated: An outcome in dystrophinopathy.
Muscle Nerve
; 52(3): 356-62, 2015 Sep.
Artigo
em Inglês
| MEDLINE | ID: mdl-25641021
17.
Outcome reliability in non-ambulatory boys/men with Duchenne muscular dystrophy.
Muscle Nerve
; 51(4): 522-32, 2015 Apr.
Artigo
em Inglês
| MEDLINE | ID: mdl-25056178
18.
Validity of remote live stream video evaluation of the North Star Ambulatory Assessment in patients with Duchenne muscular dystrophy.
PLoS One
; 19(5): e0300700, 2024.
Artigo
em Inglês
| MEDLINE | ID: mdl-38753764
19.
Feasibility and utility of in-home body weight support harness system use in young children treated for spinal muscular atrophy: A single-arm prospective cohort study.
PLoS One
; 19(3): e0300244, 2024.
Artigo
em Inglês
| MEDLINE | ID: mdl-38502672
20.
Gene therapy with bidridistrogene xeboparvovec for limb-girdle muscular dystrophy type 2E/R4: phase 1/2 trial results.
Nat Med
; 30(1): 199-206, 2024 Jan.
Artigo
em Inglês
| MEDLINE | ID: mdl-38177855