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Items: 35

1.

Towards regulatory endorsement of drug development tools to promote the application of model-informed drug development in Duchenne muscular dystrophy.

Conrado DJ, Larkindale J, Berg A, Hill M, Burton J, Abrams KR, Abresch RT, Bronson A, Chapman D, Crowther M, Duong T, Gordish-Dressman H, Harnisch L, Henricson E, Kim S, McDonald CM, Schmidt S, Vong C, Wang X, Wong BL, Yong F, Romero K; Duchenne Muscular Dystrophy Regulatory Science Consortium (D-RSC).

J Pharmacokinet Pharmacodyn. 2019 May 24. doi: 10.1007/s10928-019-09642-7. [Epub ahead of print]

PMID:
31127458
2.

Eteplirsen Treatment Attenuates Respiratory Decline in Ambulatory and Non-Ambulatory Patients with Duchenne Muscular Dystrophy.

Khan N, Eliopoulos H, Han L, Kinane TB, Lowes LP, Mendell JR, Gordish-Dressman H, Henricson EK, McDonald CM; Eteplirsen Investigators and the CINRG DNHS Investigators.

J Neuromuscul Dis. 2019;6(2):213-225. doi: 10.3233/JND-180351.

PMID:
30856119
3.

Neurodevelopmental Needs in Young Boys with Duchenne Muscular Dystrophy (DMD): Observations from the Cooperative International Neuromuscular Research Group (CINRG) DMD Natural History Study (DNHS).

Thangarajh M, Spurney CF, Gordish-Dressman H, Clemens PR, Hoffman EP, McDonald CM, Henricson EK, Investigators C.

PLoS Curr. 2018 Oct 17;10. pii: ecurrents.md.4cdeb6970e54034db2bc3dfa54b4d987. doi: 10.1371/currents.md.4cdeb6970e54034db2bc3dfa54b4d987.

4.

Longitudinal pulmonary function testing outcome measures in Duchenne muscular dystrophy: Long-term natural history with and without glucocorticoids.

McDonald CM, Gordish-Dressman H, Henricson EK, Duong T, Joyce NC, Jhawar S, Leinonen M, Hsu F, Connolly AM, Cnaan A, Abresch RT; CINRG investigators for PubMed.

Neuromuscul Disord. 2018 Nov;28(11):897-909. doi: 10.1016/j.nmd.2018.07.004. Epub 2018 Aug 29.

5.

Long-term effects of glucocorticoids on function, quality of life, and survival in patients with Duchenne muscular dystrophy: a prospective cohort study.

McDonald CM, Henricson EK, Abresch RT, Duong T, Joyce NC, Hu F, Clemens PR, Hoffman EP, Cnaan A, Gordish-Dressman H; CINRG Investigators.

Lancet. 2018 Feb 3;391(10119):451-461. doi: 10.1016/S0140-6736(17)32160-8. Epub 2017 Nov 22.

PMID:
29174484
6.

Pulmonary Endpoints in Duchenne Muscular Dystrophy. A Workshop Summary.

Finder J, Mayer OH, Sheehan D, Sawnani H, Abresch RT, Benditt J, Birnkrant DJ, Duong T, Henricson E, Kinnett K, McDonald CM, Connolly AM.

Am J Respir Crit Care Med. 2017 Aug 15;196(4):512-519. doi: 10.1164/rccm.201703-0507WS. Review.

PMID:
28636407
7.

Duchenne Regulatory Science Consortium Meeting on Disease Progression Modeling for Duchenne Muscular Dystrophy.

Larkindale J, Abresch R, Aviles E, Bronson A, Chin J, Furlong P, Gordish-Dressman H, Habeeb-Louks E, Henricson E, Kroger H, Lynn C, Lynn S, Martin D, Nuckolls G, Rooney W, Romero K, Sweeney L, Vandenborne K, Walter G, Wolff J, Wong B, McDonald CM, Duchenne Regulatory Science Consortium Imaging-Dmd Consortium And The Cinrg Investigators MO.

PLoS Curr. 2017 Jan 12;9. pii: ecurrents.md.83071bbd728982f2f1073f4950e03586. doi: 10.1371/currents.md.83071bbd728982f2f1073f4950e03586.

8.

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.

9.

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.

10.

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.

11.

Prednisone/prednisolone and deflazacort regimens in the CINRG Duchenne Natural History Study.

Bello L, Gordish-Dressman H, Morgenroth LP, Henricson EK, Duong T, Hoffman EP, Cnaan A, McDonald CM; CINRG Investigators.

Neurology. 2015 Sep 22;85(12):1048-55. doi: 10.1212/WNL.0000000000001950. Epub 2015 Aug 26.

12.

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.

13.

Genetic modifiers of ambulation in the Cooperative International Neuromuscular Research Group Duchenne Natural History Study.

Bello L, Kesari A, Gordish-Dressman H, Cnaan A, Morgenroth LP, Punetha J, Duong T, Henricson EK, Pegoraro E, McDonald CM, Hoffman EP; Cooperative International Neuromuscular Research Group Investigators.

Ann Neurol. 2015 Apr;77(4):684-96. doi: 10.1002/ana.24370. Epub 2015 Mar 13.

14.

Ataluren treatment of patients with nonsense mutation dystrophinopathy.

Bushby K, Finkel R, Wong B, Barohn R, Campbell C, Comi GP, Connolly AM, Day JW, Flanigan KM, Goemans N, Jones KJ, Mercuri E, Quinlivan R, Renfroe JB, Russman B, Ryan MM, Tulinius M, Voit T, Moore SA, Lee Sweeney H, Abresch RT, Coleman KL, Eagle M, Florence J, Gappmaier E, Glanzman AM, Henricson E, Barth J, Elfring GL, Reha A, Spiegel RJ, O'donnell MW, Peltz SW, Mcdonald CM; PTC124-GD-007-DMD STUDY GROUP.

Muscle Nerve. 2014 Oct;50(4):477-87. doi: 10.1002/mus.24332.

15.

Discovery of serum protein biomarkers in the mdx mouse model and cross-species comparison to Duchenne muscular dystrophy patients.

Hathout Y, Marathi RL, Rayavarapu S, Zhang A, Brown KJ, Seol H, Gordish-Dressman H, Cirak S, Bello L, Nagaraju K, Partridge T, Hoffman EP, Takeda S, Mah JK, Henricson E, McDonald C.

Hum Mol Genet. 2014 Dec 15;23(24):6458-69. doi: 10.1093/hmg/ddu366. Epub 2014 Jul 15.

16.

The 6-Minute Walk Test and Person-Reported Outcomes in Boys with Duchenne Muscular Dystrophy and Typically Developing Controls: Longitudinal Comparisons and Clinically-Meaningful Changes Over One Year.

Henricson E, Abresch R, Han JJ, Nicorici A, Goude Keller E, de Bie E, McDonald CM.

Version 3. PLoS Curr. 2013 Jul 8 [revised 2013 Jan 1];5. pii: ecurrents.md.9e17658b007eb79fcd6f723089f79e06. doi: 10.1371/currents.md.9e17658b007eb79fcd6f723089f79e06.

17.

The 6-minute walk test and other endpoints in Duchenne muscular dystrophy: longitudinal natural history observations over 48 weeks from a multicenter study.

McDonald CM, Henricson EK, Abresch RT, Florence JM, Eagle M, Gappmaier E, Glanzman AM; PTC124-GD-007-DMD Study Group, Spiegel R, Barth J, Elfring G, Reha A, Peltz S.

Muscle Nerve. 2013 Sep;48(3):343-56. doi: 10.1002/mus.23902. Epub 2013 Jun 26.

18.

The cooperative international neuromuscular research group Duchenne natural history study--a longitudinal investigation in the era of glucocorticoid therapy: design of protocol and the methods used.

McDonald CM, Henricson EK, Abresch RT, Han JJ, Escolar DM, Florence JM, Duong T, Arrieta A, Clemens PR, Hoffman EP, Cnaan A; Cinrg Investigators.

Muscle Nerve. 2013 Jul;48(1):32-54. doi: 10.1002/mus.23807. Epub 2013 May 16.

19.

The 6-minute walk test and other clinical endpoints in duchenne muscular dystrophy: reliability, concurrent validity, and minimal clinically important differences from a multicenter study.

McDonald CM, Henricson EK, Abresch RT, Florence J, Eagle M, Gappmaier E, Glanzman AM; PTC124-GD-007-DMD Study Group, Spiegel R, Barth J, Elfring G, Reha A, Peltz SW.

Muscle Nerve. 2013 Sep;48(3):357-68. doi: 10.1002/mus.23905. Epub 2013 Jul 17.

20.

The cooperative international neuromuscular research group Duchenne natural history study: glucocorticoid treatment preserves clinically meaningful functional milestones and reduces rate of disease progression as measured by manual muscle testing and other commonly used clinical trial outcome measures.

Henricson EK, Abresch RT, Cnaan A, Hu F, Duong T, Arrieta A, Han J, Escolar DM, Florence JM, Clemens PR, Hoffman EP, McDonald CM; CINRG Investigators.

Muscle Nerve. 2013 Jul;48(1):55-67. doi: 10.1002/mus.23808. Epub 2013 May 6.

21.

Randomized controlled trial comparing acupuncture with placebo acupuncture for the treatment of carpal tunnel syndrome.

Yao E, Gerritz PK, Henricson E, Abresch T, Kim J, Han J, Wang K, Zhao H.

PM R. 2012 May;4(5):367-73. doi: 10.1016/j.pmrj.2012.01.008. Epub 2012 Mar 7.

PMID:
22405683
22.

Pentoxifylline as a rescue treatment for DMD: a randomized double-blind clinical trial.

Escolar DM, Zimmerman A, Bertorini T, Clemens PR, Connolly AM, Mesa L, Gorni K, Kornberg A, Kolski H, Kuntz N, Nevo Y, Tesi-Rocha C, Nagaraju K, Rayavarapu S, Hache LP, Mayhew JE, Florence J, Hu F, Arrieta A, Henricson E, Leshner RT, Mah JK.

Neurology. 2012 Mar 20;78(12):904-13. doi: 10.1212/WNL.0b013e31824c46be. Epub 2012 Mar 7.

23.

Percent-predicted 6-minute walk distance in duchenne muscular dystrophy to account for maturational influences.

Henricson E, Abresch R, Han JJ, Nicorici A, Goude Keller E, Elfring G, Reha A, Barth J, McDonald CM.

Version 2. PLoS Curr. 2012 Jan 25 [revised 2012 Jan 1];4:RRN1297.

24.

Quality-of-life measures in children with neurological conditions: pediatric Neuro-QOL.

Lai JS, Nowinski C, Victorson D, Bode R, Podrabsky T, McKinney N, Straube D, Holmes GL, McDonald CM, Henricson E, Abresch RT, Moy CS, Cella D.

Neurorehabil Neural Repair. 2012 Jan;26(1):36-47. doi: 10.1177/1545968311412054. Epub 2011 Jul 25.

25.

Randomized, blinded trial of weekend vs daily prednisone in Duchenne muscular dystrophy.

Escolar DM, Hache LP, Clemens PR, Cnaan A, McDonald CM, Viswanathan V, Kornberg AJ, Bertorini TE, Nevo Y, Lotze T, Pestronk A, Ryan MM, Monasterio E, Day JW, Zimmerman A, Arrieta A, Henricson E, Mayhew J, Florence J, Hu F, Connolly AM.

Neurology. 2011 Aug 2;77(5):444-52. doi: 10.1212/WNL.0b013e318227b164. Epub 2011 Jul 13.

26.

CINRG pilot trial of coenzyme Q10 in steroid-treated Duchenne muscular dystrophy.

Spurney CF, Rocha CT, Henricson E, Florence J, Mayhew J, Gorni K, Pasquali L, Pestronk A, Martin GR, Hu F, Nie L, Connolly AM, Escolar DM; Cooperative International Neuromuscular Research Group Investigators.

Muscle Nerve. 2011 Aug;44(2):174-8. doi: 10.1002/mus.22047. Epub 2011 Jun 22.

27.

Liquid formulation of pentoxifylline is a poorly tolerated treatment for duchenne dystrophy.

Zimmerman A, Clemens PR, Tesi-Rocha C, Connolly A, Iannaccone ST, Kuntz N, Arrieta A, Hache L, Henricson E, Hu F, Mayhew J, Escolar DM.

Muscle Nerve. 2011 Aug;44(2):170-3. doi: 10.1002/mus.22127. Epub 2011 Jun 14.

28.

The 6-minute walk test in Duchenne/Becker muscular dystrophy: longitudinal observations.

McDonald CM, Henricson EK, Han JJ, Abresch RT, Nicorici A, Atkinson L, Elfring GL, Reha A, Miller LL.

Muscle Nerve. 2010 Dec;42(6):966-74. doi: 10.1002/mus.21808.

PMID:
21038378
29.

Relationship between clinical outcome measures and parent proxy reports of health-related quality of life in ambulatory children with Duchenne muscular dystrophy.

McDonald CM, McDonald DA, Bagley A, Sienko Thomas S, Buckon CE, Henricson E, Nicorici A, Sussman MD.

J Child Neurol. 2010 Sep;25(9):1130-44. doi: 10.1177/0883073810371509. Epub 2010 Jun 17.

30.

The 6-minute walk test as a new outcome measure in Duchenne muscular dystrophy.

McDonald CM, Henricson EK, Han JJ, Abresch RT, Nicorici A, Elfring GL, Atkinson L, Reha A, Hirawat S, Miller LL.

Muscle Nerve. 2010 Apr;41(4):500-10. doi: 10.1002/mus.21544.

PMID:
19941337
31.

CINRG pilot trial of oxatomide in steroid-naïve Duchenne muscular dystrophy.

Buyse GM, Goemans N, Henricson E, Jara A, van den Hauwe M, Leshner R, Florence JM, Mayhew JE, Escolar DM.

Eur J Paediatr Neurol. 2007 Nov;11(6):337-40. Epub 2007 Apr 24.

PMID:
17459739
32.

Reliable surrogate outcome measures in multicenter clinical trials of Duchenne muscular dystrophy.

Mayhew JE, Florence JM, Mayhew TP, Henricson EK, Leshner RT, McCarter RJ, Escolar DM.

Muscle Nerve. 2007 Jan;35(1):36-42.

PMID:
16969838
33.

CINRG randomized controlled trial of creatine and glutamine in Duchenne muscular dystrophy.

Escolar DM, Buyse G, Henricson E, Leshner R, Florence J, Mayhew J, Tesi-Rocha C, Gorni K, Pasquali L, Patel KM, McCarter R, Huang J, Mayhew T, Bertorini T, Carlo J, Connolly AM, Clemens PR, Goemans N, Iannaccone ST, Igarashi M, Nevo Y, Pestronk A, Subramony SH, Vedanarayanan VV, Wessel H; CINRG Group.

Ann Neurol. 2005 Jul;58(1):151-5.

PMID:
15984021
34.

Collaborative translational research leading to multicenter clinical trials in Duchenne muscular dystrophy: the Cooperative International Neuromuscular Research Group (CINRG).

Escolar DM, Henricson EK, Pasquali L, Gorni K, Hoffman EP.

Neuromuscul Disord. 2002 Oct;12 Suppl 1:S147-154. Review.

PMID:
12206809
35.

Clinical evaluator reliability for quantitative and manual muscle testing measures of strength in children.

Escolar DM, Henricson EK, Mayhew J, Florence J, Leshner R, Patel KM, Clemens PR.

Muscle Nerve. 2001 Jun;24(6):787-93.

PMID:
11360262

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