Detalhe da pesquisa
1.
Small Molecule Targets TMED9 and Promotes Lysosomal Degradation to Reverse Proteinopathy.
Cell
; 178(3): 521-535.e23, 2019 07 25.
Artigo
em Inglês
| MEDLINE | ID: mdl-31348885
2.
A Next Generation Connectivity Map: L1000 Platform and the First 1,000,000 Profiles.
Cell
; 171(6): 1437-1452.e17, 2017 Nov 30.
Artigo
em Inglês
| MEDLINE | ID: mdl-29195078
3.
Binding of TMPRSS2-ERG to BAF Chromatin Remodeling Complexes Mediates Prostate Oncogenesis.
Mol Cell
; 71(4): 554-566.e7, 2018 08 16.
Artigo
em Inglês
| MEDLINE | ID: mdl-30078722
4.
Quantitative muscle analysis in facioscapulohumeral muscular dystrophy using whole-body fat-referenced MRI: Protocol development, multicenter feasibility, and repeatability.
Muscle Nerve
; 66(2): 183-192, 2022 08.
Artigo
em Inglês
| MEDLINE | ID: mdl-35585766
5.
Phase 1 clinical trial of losmapimod in facioscapulohumeral dystrophy: Safety, tolerability, pharmacokinetics, and target engagement.
Br J Clin Pharmacol
; 87(12): 4658-4669, 2021 12.
Artigo
em Inglês
| MEDLINE | ID: mdl-33931884
6.
p38α Regulates Expression of DUX4 in a Model of Facioscapulohumeral Muscular Dystrophy.
J Pharmacol Exp Ther
; 374(3): 489-498, 2020 09.
Artigo
em Inglês
| MEDLINE | ID: mdl-32576599
7.
Discovery of heterocyclic sulfonamides as sphingosine 1-phosphate receptor 1 (S1P1) antagonists.
Bioorg Med Chem Lett
; 25(10): 2041-5, 2015.
Artigo
em Inglês
| MEDLINE | ID: mdl-25890801
8.
Safety and efficacy of losmapimod in facioscapulohumeral muscular dystrophy (ReDUX4): a randomised, double-blind, placebo-controlled phase 2b trial.
Lancet Neurol
; 23(5): 477-486, 2024 May.
Artigo
em Inglês
| MEDLINE | ID: mdl-38631764
9.
Social communication in fragile X syndrome: pilot examination of the Brief Observation of Social Communication Change (BOSCC).
J Neurodev Disord
; 14(1): 4, 2022 01 08.
Artigo
em Inglês
| MEDLINE | ID: mdl-35034602
10.
The feasibility and utility of hair follicle sampling to measure FMRP and FMR1 mRNA in children with or without fragile X syndrome: a pilot study.
J Neurodev Disord
; 14(1): 57, 2022 12 09.
Artigo
em Inglês
| MEDLINE | ID: mdl-36494616
11.
Quantitative Muscle Analysis in FSHD Using Whole-Body Fat-Referenced MRI: Composite Scores for Longitudinal and Cross-sectional Analysis.
Neurology
; 99(9): e877-e889, 2022 08 30.
Artigo
em Inglês
| MEDLINE | ID: mdl-35750498
12.
FMRP Levels in Human Peripheral Blood Leukocytes Correlates with Intellectual Disability.
Diagnostics (Basel)
; 11(10)2021 Sep 28.
Artigo
em Inglês
| MEDLINE | ID: mdl-34679478
13.
A High Content Screen for Mucin-1-Reducing Compounds Identifies Fostamatinib as a Candidate for Rapid Repurposing for Acute Lung Injury during the COVID-19 pandemic.
bioRxiv
; 2020 Jun 30.
Artigo
em Inglês
| MEDLINE | ID: mdl-32637960
14.
Development and Validation of a Mass Spectrometry-Based Assay for the Molecular Diagnosis of Mucin-1 Kidney Disease.
J Mol Diagn
; 18(4): 566-71, 2016 07.
Artigo
em Inglês
| MEDLINE | ID: mdl-27157321
15.
A small molecule that binds and inhibits the ETV1 transcription factor oncoprotein.
Mol Cancer Ther
; 13(6): 1492-502, 2014 Jun.
Artigo
em Inglês
| MEDLINE | ID: mdl-24737027
16.
Discovery of (+)-N-(3-aminopropyl)-N-[1-(5-benzyl-3-methyl-4-oxo-[1,2]thiazolo[5,4-d]pyrimidin-6-yl)-2-methylpropyl]-4-methylbenzamide (AZD4877), a kinesin spindle protein inhibitor and potential anticancer agent.
J Med Chem
; 54(19): 6734-50, 2011 Oct 13.
Artigo
em Inglês
| MEDLINE | ID: mdl-21899292