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Items: 1 to 20 of 85

1.

Phase IIa trial in Duchenne muscular dystrophy shows vamorolone is a first-in-class dissociative steroidal anti-inflammatory drug.

Conklin LS, Damsker JM, Hoffman EP, Jusko WJ, Mavroudis PD, Schwartz BD, Mengle-Gaw LJ, Smith EC, Mah JK, Guglieri M, Nevo Y, Kuntz N, McDonald CM, Tulinius M, Ryan MM, Webster R, Castro D, Finkel RS, Smith AL, Morgenroth LP, Arrieta A, Shimony M, Jaros M, Shale P, McCall JM, Hathout Y, Nagaraju K, van den Anker J, Ward LM, Ahmet A, Cornish MR, Clemens PR.

Pharmacol Res. 2018 Sep 13;136:140-150. doi: 10.1016/j.phrs.2018.09.007. [Epub ahead of print]

2.

Author Correction: Myoblasts and macrophages are required for therapeutic morpholino antisense oligonucleotide delivery to dystrophic muscle.

Novak JS, Hogarth MW, Boehler JF, Nearing M, Vila MC, Heredia R, Fiorillo AA, Zhang A, Hathout Y, Hoffman EP, Jaiswal JK, Nagaraju K, Cirak S, Partridge TA.

Nat Commun. 2018 Mar 23;9(1):1256. doi: 10.1038/s41467-018-03709-8.

3.

Author Correction: Myoblasts and macrophages are required for therapeutic morpholino antisense oligonucleotide delivery to dystrophic muscle.

Novak JS, Hogarth MW, Boehler JF, Nearing M, Vila MC, Heredia R, Fiorillo AA, Zhang A, Hathout Y, Hoffman EP, Jaiswal JK, Nagaraju K, Cirak S, Partridge TA.

Nat Commun. 2018 Jan 15;9(1):208. doi: 10.1038/s41467-017-02206-8.

4.

Myoblasts and macrophages are required for therapeutic morpholino antisense oligonucleotide delivery to dystrophic muscle.

Novak JS, Hogarth MW, Boehler JF, Nearing M, Vila MC, Heredia R, Fiorillo AA, Zhang A, Hathout Y, Hoffman EP, Jaiswal JK, Nagaraju K, Cirak S, Partridge TA.

Nat Commun. 2017 Oct 16;8(1):941. doi: 10.1038/s41467-017-00924-7. Erratum in: Nat Commun. 2018 Jan 15;9(1):208. Nat Commun. 2018 Mar 23;9(1):1256.

5.

Interleukin 1 Receptor-Like 1 Protein (ST2) is a Potential Biomarker for Cardiomyopathy in Duchenne Muscular Dystrophy.

Anderson J, Seol H, Gordish-Dressman H, Hathout Y, Spurney CF; CINRG Investigators.

Pediatr Cardiol. 2017 Dec;38(8):1606-1612. doi: 10.1007/s00246-017-1703-9. Epub 2017 Aug 18.

PMID:
28821969
6.

Facilitating orphan drug development: Proceedings of the TREAT-NMD International Conference, December 2015, Washington, DC, USA.

Hoffman EP; Workshop Participants; TREAT-NMD Alliance.

Neuromuscul Disord. 2017 Jul;27(7):693-701. doi: 10.1016/j.nmd.2017.02.013. Epub 2017 Mar 4. No abstract available.

PMID:
28434909
7.

Mitochondria mediate cell membrane repair and contribute to Duchenne muscular dystrophy.

Vila MC, Rayavarapu S, Hogarth MW, Van der Meulen JH, Horn A, Defour A, Takeda S, Brown KJ, Hathout Y, Nagaraju K, Jaiswal JK.

Cell Death Differ. 2017 Feb;24(2):330-342. doi: 10.1038/cdd.2016.127. Epub 2016 Nov 11.

8.

PARP1 Differentially Interacts with Promoter region of DUX4 Gene in FSHD Myoblasts.

Sharma V, Pandey SN, Khawaja H, Brown KJ, Hathout Y, Chen YW.

J Genet Syndr Gene Ther. 2016 Aug;7(4). pii: 303. Epub 2016 Aug 8.

9.

Identification of Pathway-Specific Serum Biomarkers of Response to Glucocorticoid and Infliximab Treatment in Children with Inflammatory Bowel Disease.

Heier CR, Fiorillo AA, Chaisson E, Gordish-Dressman H, Hathout Y, Damsker JM, Hoffman EP, Conklin LS.

Clin Transl Gastroenterol. 2016 Sep 15;7(9):e192. doi: 10.1038/ctg.2016.49.

10.

Serum pharmacodynamic biomarkers for chronic corticosteroid treatment of children.

Hathout Y, Conklin LS, Seol H, Gordish-Dressman H, Brown KJ, Morgenroth LP, Nagaraju K, Heier CR, Damsker JM, van den Anker JN, Henricson E, Clemens PR, Mah JK, McDonald C, Hoffman EP.

Sci Rep. 2016 Aug 17;6:31727. doi: 10.1038/srep31727.

11.

Correction: Discovery of Metabolic Biomarkers for Duchenne Muscular Dystrophy within a Natural History Study.

Boca SM, Nishida M, Harris M, Rao S, Cheema AK, Gill K, Wang D, An L, Gauba R, Seol H, Morgenroth LP, Henricson E, McDonald C, Mah JK, Clemens PR, Hoffman EP, Hathout Y, Madhavan S.

PLoS One. 2016 Jul 19;11(7):e0159895. doi: 10.1371/journal.pone.0159895. eCollection 2016.

12.

Discovery of Metabolic Biomarkers for Duchenne Muscular Dystrophy within a Natural History Study.

Boca SM, Nishida M, Harris M, Rao S, Cheema AK, Gill K, Seol H, Morgenroth LP, Henricson E, McDonald C, Mah JK, Clemens PR, Hoffman EP, Hathout Y, Madhavan S.

PLoS One. 2016 Apr 15;11(4):e0153461. doi: 10.1371/journal.pone.0153461. eCollection 2016. Erratum in: PLoS One. 2016;11(7):e0159895.

13.

Clinical utility of serum biomarkers in Duchenne muscular dystrophy.

Hathout Y, Seol H, Han MH, Zhang A, Brown KJ, Hoffman EP.

Clin Proteomics. 2016 Apr 5;13:9. doi: 10.1186/s12014-016-9109-x. eCollection 2016. Review.

14.

Elusive sources of variability of dystrophin rescue by exon skipping.

Vila MC, Klimek MB, Novak JS, Rayavarapu S, Uaesoontrachoon K, Boehler JF, Fiorillo AA, Hogarth MW, Zhang A, Shaughnessy C, Gordish-Dressman H, Burki U, Straub V, Lu QL, Partridge TA, Brown KJ, Hathout Y, van den Anker J, Hoffman EP, Nagaraju K.

Skelet Muscle. 2015 Dec 1;5:44. doi: 10.1186/s13395-015-0070-6. eCollection 2015.

15.

The use of urinary and kidney SILAM proteomics to monitor kidney response to high dose morpholino oligonucleotides in the mdx mouse.

Zhang A, Uaesoontrachoon K, Shaughnessy C, Das JR, Rayavarapu S, Brown KJ, Ray PE, Nagaraju K, van den Anker JN, Hoffman EP, Hathout Y.

Toxicol Rep. 2015;2:838-849.

16.

Proteomic methods for biomarker discovery and validation. Are we there yet?

Hathout Y.

Expert Rev Proteomics. 2015 Aug;12(4):329-31. doi: 10.1586/14789450.2015.1064771.

PMID:
26186709
17.

Large-scale serum protein biomarker discovery in Duchenne muscular dystrophy.

Hathout Y, Brody E, Clemens PR, Cripe L, DeLisle RK, Furlong P, Gordish-Dressman H, Hache L, Henricson E, Hoffman EP, Kobayashi YM, Lorts A, Mah JK, McDonald C, Mehler B, Nelson S, Nikrad M, Singer B, Steele F, Sterling D, Sweeney HL, Williams S, Gold L.

Proc Natl Acad Sci U S A. 2015 Jun 9;112(23):7153-8. doi: 10.1073/pnas.1507719112. Epub 2015 May 26.

18.

Urinary biomarkers of kidney diseases in HIV-infected children.

Perazzo S, Soler-García ÁA, Hathout Y, Das JR, Ray PE.

Proteomics Clin Appl. 2015 Jun;9(5-6):490-500. doi: 10.1002/prca.201400193. Review.

19.

miR-184 regulates ezrin, LAMP-1 expression, affects phagocytosis in human retinal pigment epithelium and is downregulated in age-related macular degeneration.

Murad N, Kokkinaki M, Gunawardena N, Gunawan MS, Hathout Y, Janczura KJ, Theos AC, Golestaneh N.

FEBS J. 2014 Dec;281(23):5251-64. doi: 10.1111/febs.13066. Epub 2014 Oct 13.

20.

Multi-omic integrated networks connect DNA methylation and miRNA with skeletal muscle plasticity to chronic exercise in Type 2 diabetic obesity.

Rowlands DS, Page RA, Sukala WR, Giri M, Ghimbovschi SD, Hayat I, Cheema BS, Lys I, Leikis M, Sheard PW, Wakefield SJ, Breier B, Hathout Y, Brown K, Marathi R, Orkunoglu-Suer FE, Devaney JM, Leiken B, Many G, Krebs J, Hopkins WG, Hoffman EP.

Physiol Genomics. 2014 Oct 15;46(20):747-65. doi: 10.1152/physiolgenomics.00024.2014. Epub 2014 Aug 19.

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