Detalhe da pesquisa
1.
Delandistrogene Moxeparvovec Gene Therapy in Ambulatory Patients (Aged ≥4 to <8 Years) with Duchenne Muscular Dystrophy: 1-Year Interim Results from Study SRP-9001-103 (ENDEAVOR).
Ann Neurol
; 94(5): 955-968, 2023 11.
Artigo
em Inglês
| MEDLINE | ID: mdl-37539981
2.
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
3.
Therapeutic microRNA delivery suppresses tumorigenesis in a murine liver cancer model.
Cell
; 137(6): 1005-17, 2009 Jun 12.
Artigo
em Inglês
| MEDLINE | ID: mdl-19524505
4.
AAV1.NT-3 gene therapy for X-linked Charcot-Marie-Tooth neuropathy type 1.
Gene Ther
; 29(3-4): 127-137, 2022 04.
Artigo
em Inglês
| MEDLINE | ID: mdl-33542455
5.
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
6.
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
7.
Current Clinical Applications of In Vivo Gene Therapy with AAVs.
Mol Ther
; 29(2): 464-488, 2021 02 03.
Artigo
em Inglês
| MEDLINE | ID: mdl-33309881
8.
Single-Dose Gene-Replacement Therapy for Spinal Muscular Atrophy.
N Engl J Med
; 377(18): 1713-1722, 2017 11 02.
Artigo
em Inglês
| MEDLINE | ID: mdl-29091557
9.
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
10.
Contributions from medical geneticists in clinical trials of genetic therapies: A points to consider statement of the American College of Medical Genetics and Genomics (ACMG).
Genet Med
; 25(6): 100831, 2023 06.
Artigo
em Inglês
| MEDLINE | ID: mdl-37031408
11.
Systemic AAV-Mediated ß-Sarcoglycan Delivery Targeting Cardiac and Skeletal Muscle Ameliorates Histological and Functional Deficits in LGMD2E Mice.
Mol Ther
; 25(4): 855-869, 2017 04 05.
Artigo
em Inglês
| MEDLINE | ID: mdl-28284983
12.
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
13.
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
14.
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
15.
Therapy for Spinal Muscular Atrophy.
N Engl J Med
; 378(5): 487, 2018 02 01.
Artigo
em Inglês
| MEDLINE | ID: mdl-29394473
16.
Clinical phenotypes as predictors of the outcome of skipping around DMD exon 45.
Ann Neurol
; 77(4): 668-74, 2015 Apr.
Artigo
em Inglês
| MEDLINE | ID: mdl-25612243
17.
Clinical Follow-Up for Duchenne Muscular Dystrophy Newborn Screening: A Proposal.
Muscle Nerve
; 54(2): 186-91, 2016 08.
Artigo
em Inglês
| MEDLINE | ID: mdl-27170260
18.
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
19.
Improving single injection CSF delivery of AAV9-mediated gene therapy for SMA: a dose-response study in mice and nonhuman primates.
Mol Ther
; 23(3): 477-87, 2015 Mar.
Artigo
em Inglês
| MEDLINE | ID: mdl-25358252
20.
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