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.
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
3.
Comparison of strength testing modalities in dysferlinopathy.
Muscle Nerve
; 66(2): 159-166, 2022 08.
Artigo
em Inglês
| MEDLINE | ID: mdl-35506767
4.
Reliability and construct validity of the Duchenne Video Assessment.
Muscle Nerve
; 64(2): 180-189, 2021 08.
Artigo
em Inglês
| MEDLINE | ID: mdl-34050939
5.
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
6.
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
7.
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
8.
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
9.
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
10.
'Learn From Every Patient': implementation and early results of a learning health system.
Dev Med Child Neurol
; 59(2): 183-191, 2017 Feb.
Artigo
em Inglês
| MEDLINE | ID: mdl-27545839
11.
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
12.
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
13.
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
14.
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
15.
Eteplirsen for the treatment of Duchenne muscular dystrophy.
Ann Neurol
; 74(5): 637-47, 2013 Nov.
Artigo
em Inglês
| MEDLINE | ID: mdl-23907995
16.
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
17.
Increase in Full-Length Dystrophin by Exon Skipping in Duchenne Muscular Dystrophy Patients with Single Exon Duplications: An Open-label Study.
J Neuromuscul Dis
; 11(3): 679-685, 2024.
Artigo
em Inglês
| MEDLINE | ID: mdl-38461513
18.
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
19.
Correlation of knee strength to functional outcomes in Becker muscular dystrophy.
Muscle Nerve
; 47(4): 550-4, 2013 Apr.
Artigo
em Inglês
| MEDLINE | ID: mdl-23460325
20.
Home-based video assessment of ease of movement for patients with Duchenne: Interviews with physical therapists to select movement tasks.
Physiother Res Int
; : e1993, 2023 Jan 30.
Artigo
em Inglês
| MEDLINE | ID: mdl-36718112