Detalhe da pesquisa
1.
A mouse model of inherited choline kinase ß-deficiency presents with specific cardiac abnormalities and a predisposition to arrhythmia.
J Biol Chem
; 298(3): 101716, 2022 03.
Artigo
em Inglês
| MEDLINE | ID: mdl-35151687
2.
A long-read RNA-seq approach to identify novel transcripts of very large genes.
Genome Res
; 30(6): 885-897, 2020 06.
Artigo
em Inglês
| MEDLINE | ID: mdl-32660935
3.
A novel estrogen receptor 1: sphingomyelin phosphodiesterase acid-like 3B pathway mediates rituximab response in myositis patients.
Rheumatology (Oxford)
; 62(8): 2864-2871, 2023 08 01.
Artigo
em Inglês
| MEDLINE | ID: mdl-36478205
4.
Disruption of a key ligand-H-bond network drives dissociative properties in vamorolone for Duchenne muscular dystrophy treatment.
Proc Natl Acad Sci U S A
; 117(39): 24285-24293, 2020 09 29.
Artigo
em Inglês
| MEDLINE | ID: mdl-32917814
5.
Serum biomarkers associated with baseline clinical severity in young steroid-naïve Duchenne muscular dystrophy boys.
Hum Mol Genet
; 29(15): 2481-2495, 2020 08 29.
Artigo
em Inglês
| MEDLINE | ID: mdl-32592467
6.
Sphingosine Phosphate Lyase Is Upregulated in Duchenne Muscular Dystrophy, and Its Inhibition Early in Life Attenuates Inflammation and Dystrophy in Mdx Mice.
Int J Mol Sci
; 23(14)2022 Jul 08.
Artigo
em Inglês
| MEDLINE | ID: mdl-35886926
7.
Elevation of fast but not slow troponin I in the circulation of patients with Becker and Duchenne muscular dystrophy.
Muscle Nerve
; 64(1): 43-49, 2021 07.
Artigo
em Inglês
| MEDLINE | ID: mdl-33683712
8.
A Dystrophin Exon-52 Deleted Miniature Pig Model of Duchenne Muscular Dystrophy and Evaluation of Exon Skipping.
Int J Mol Sci
; 22(23)2021 Dec 02.
Artigo
em Inglês
| MEDLINE | ID: mdl-34884867
9.
Efficacy and safety of vamorolone in Duchenne muscular dystrophy: An 18-month interim analysis of a non-randomized open-label extension study.
PLoS Med
; 17(9): e1003222, 2020 09.
Artigo
em Inglês
| MEDLINE | ID: mdl-32956407
10.
Morpholino-induced exon skipping stimulates cell-mediated and humoral responses to dystrophin in mdx mice.
J Pathol
; 248(3): 339-351, 2019 07.
Artigo
em Inglês
| MEDLINE | ID: mdl-30883742
11.
Pharmacotherapy of Duchenne Muscular Dystrophy.
Handb Exp Pharmacol
; 261: 25-37, 2020.
Artigo
em Inglês
| MEDLINE | ID: mdl-31375923
12.
Association Study of Exon Variants in the NF-κB and TGFß Pathways Identifies CD40 as a Modifier of Duchenne Muscular Dystrophy.
Am J Hum Genet
; 99(5): 1163-1171, 2016 Nov 03.
Artigo
em Inglês
| MEDLINE | ID: mdl-27745838
13.
Long-term effects of glucocorticoids on function, quality of life, and survival in patients with Duchenne muscular dystrophy: a prospective cohort study.
Lancet
; 391(10119): 451-461, 2018 02 03.
Artigo
em Inglês
| MEDLINE | ID: mdl-29174484
14.
Influence of ß2 adrenergic receptor genotype on risk of nocturnal ventilation in patients with Duchenne muscular dystrophy.
Respir Res
; 20(1): 221, 2019 Oct 16.
Artigo
em Inglês
| MEDLINE | ID: mdl-31619245
15.
Membrane Stabilization by Modified Steroid Offers a Potential Therapy for Muscular Dystrophy Due to Dysferlin Deficit.
Mol Ther
; 26(9): 2231-2242, 2018 09 05.
Artigo
em Inglês
| MEDLINE | ID: mdl-30166241
16.
Mechanisms of allelic and clinical heterogeneity of lamin A/C phenotypes.
Physiol Genomics
; 50(9): 694-704, 2018 09 01.
Artigo
em Inglês
| MEDLINE | ID: mdl-29750601
17.
Muscle miRNAome shows suppression of chronic inflammatory miRNAs with both prednisone and vamorolone.
Physiol Genomics
; 50(9): 735-745, 2018 09 01.
Artigo
em Inglês
| MEDLINE | ID: mdl-29883261
18.
Phase IIa trial in Duchenne muscular dystrophy shows vamorolone is a first-in-class dissociative steroidal anti-inflammatory drug.
Pharmacol Res
; 136: 140-150, 2018 10.
Artigo
em Inglês
| MEDLINE | ID: mdl-30219580
19.
Large-scale serum protein biomarker discovery in Duchenne muscular dystrophy.
Proc Natl Acad Sci U S A
; 112(23): 7153-8, 2015 Jun 09.
Artigo
em Inglês
| MEDLINE | ID: mdl-26039989
20.
Concerns Regarding Therapeutic Implications of Very Low-Level Dystrophin.
Ann Neurol
; 90(1): 176, 2021 07.
Artigo
em Inglês
| MEDLINE | ID: mdl-33948977