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Items: 44

1.

Barriers and facilitators to clinical trial participation among parents of children with pediatric neuromuscular disorders.

Peay HL, Biesecker BB, Wilfond BS, Jarecki J, Umstead KL, Escolar DM, Tibben A.

Clin Trials. 2018 Apr;15(2):139-148. doi: 10.1177/1740774517751118. Epub 2018 Feb 23.

2.

De novo variants in KLF7 are a potential novel cause of developmental delay/intellectual disability, neuromuscular and psychiatric symptoms.

Powis Z, Petrik I, Cohen JS, Escolar D, Burton J, van Ravenswaaij-Arts CMA, Sival DA, Stegmann APA, Kleefstra T, Pfundt R, Chikarmane R, Begtrup A, Huether R, Tang S, Shinde DN.

Clin Genet. 2018 May;93(5):1030-1038. doi: 10.1111/cge.13198. Epub 2018 Jan 25.

PMID:
29251763
3.

Myostatin inhibitor ACE-031 treatment of ambulatory boys with Duchenne muscular dystrophy: Results of a randomized, placebo-controlled clinical trial.

Campbell C, McMillan HJ, Mah JK, Tarnopolsky M, Selby K, McClure T, Wilson DM, Sherman ML, Escolar D, Attie KM.

Muscle Nerve. 2017 Apr;55(4):458-464. doi: 10.1002/mus.25268. Epub 2016 Dec 23.

PMID:
27462804
4.

"Watching time tick by…": Decision making for Duchenne muscular dystrophy trials.

Peay HL, Scharff H, Tibben A, Wilfond B, Bowie J, Johnson J, Nagaraju K, Escolar D, Piacentino J, Biesecker BB.

Contemp Clin Trials. 2016 Jan;46:1-6. doi: 10.1016/j.cct.2015.11.006. Epub 2015 Nov 9.

5.

Electrical impedance myography in Duchenne muscular dystrophy and healthy controls: A multicenter study of reliability and validity.

Zaidman CM, Wang LL, Connolly AM, Florence J, Wong BL, Parsons JA, Apkon S, Goyal N, Williams E, Escolar D, Rutkove SB, Bohorquez JL; DART-EIM Clinical Evaluators Consortium.

Muscle Nerve. 2015 Oct;52(4):592-7. doi: 10.1002/mus.24611. Epub 2015 Jul 24.

PMID:
25702806
6.

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.

7.

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.

8.

Arginine butyrate: a therapeutic candidate for Duchenne muscular dystrophy.

Vianello S, Yu H, Voisin V, Haddad H, He X, Foutz AS, Sebrié C, Gillet B, Roulot M, Fougerousse F, Perronnet C, Vaillend C, Matecki S, Escolar D, Bossi L, Israël M, de la Porte S.

FASEB J. 2013 Jun;27(6):2256-69. doi: 10.1096/fj.12-215723. Epub 2013 Feb 21.

PMID:
23430975
9.

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.

10.

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.

11.

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.

12.

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.

13.

Congenital muscular dystrophies.

Sparks SE, Escolar DM.

Handb Clin Neurol. 2011;101:47-79. doi: 10.1016/B978-0-08-045031-5.00004-9. Review.

PMID:
21496624
14.

Pediatric sciatic neuropathies: a 30-year prospective study.

Srinivasan J, Ryan MM, Escolar DM, Darras B, Jones HR.

Neurology. 2011 Mar 15;76(11):976-80. doi: 10.1212/WNL.0b013e3182104394.

15.

Novel LMNA mutations in patients with Emery-Dreifuss muscular dystrophy and functional characterization of four LMNA mutations.

Scharner J, Brown CA, Bower M, Iannaccone ST, Khatri IA, Escolar D, Gordon E, Felice K, Crowe CA, Grosmann C, Meriggioli MN, Asamoah A, Gordon O, Gnocchi VF, Ellis JA, Mendell JR, Zammit PS.

Hum Mutat. 2011 Feb;32(2):152-67. doi: 10.1002/humu.21361. Epub 2011 Jan 25.

PMID:
20848652
16.

Functional and molecular effects of arginine butyrate and prednisone on muscle and heart in the mdx mouse model of Duchenne Muscular Dystrophy.

Guerron AD, Rawat R, Sali A, Spurney CF, Pistilli E, Cha HJ, Pandey GS, Gernapudi R, Francia D, Farajian V, Escolar DM, Bossi L, Becker M, Zerr P, de la Porte S, Gordish-Dressman H, Partridge T, Hoffman EP, Nagaraju K.

PLoS One. 2010 Jun 21;5(6):e11220. doi: 10.1371/journal.pone.0011220.

17.

A randomized study of alglucosidase alfa in late-onset Pompe's disease.

van der Ploeg AT, Clemens PR, Corzo D, Escolar DM, Florence J, Groeneveld GJ, Herson S, Kishnani PS, Laforet P, Lake SL, Lange DJ, Leshner RT, Mayhew JE, Morgan C, Nozaki K, Park DJ, Pestronk A, Rosenbloom B, Skrinar A, van Capelle CI, van der Beek NA, Wasserstein M, Zivkovic SA.

N Engl J Med. 2010 Apr 15;362(15):1396-406. doi: 10.1056/NEJMoa0909859.

18.

Clinical features of late-onset Pompe disease: a prospective cohort study.

Wokke JH, Escolar DM, Pestronk A, Jaffe KM, Carter GT, van den Berg LH, Florence JM, Mayhew J, Skrinar A, Corzo D, Laforet P.

Muscle Nerve. 2008 Oct;38(4):1236-45. doi: 10.1002/mus.21025.

PMID:
18816591
19.

Pediatric sciatic neuropathies due to unusual vascular causes.

Srinivasan J, Escolar D, Ryan M, Darras B, Jones HR.

J Child Neurol. 2008 Jul;23(7):738-41. doi: 10.1177/0883073808314163.

PMID:
18658074
20.

A phase I/IItrial of MYO-029 in adult subjects with muscular dystrophy.

Wagner KR, Fleckenstein JL, Amato AA, Barohn RJ, Bushby K, Escolar DM, Flanigan KM, Pestronk A, Tawil R, Wolfe GI, Eagle M, Florence JM, King WM, Pandya S, Straub V, Juneau P, Meyers K, Csimma C, Araujo T, Allen R, Parsons SA, Wozney JM, Lavallie ER, Mendell JR.

Ann Neurol. 2008 May;63(5):561-71. doi: 10.1002/ana.21338.

PMID:
18335515
21.

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
22.

The color space of foods: virgin olive oil.

Escolar D, Haro MR, Ayuso J.

J Agric Food Chem. 2007 Mar 21;55(6):2085-93. Epub 2007 Feb 17.

PMID:
17305358
23.

Challenges in drug development for muscle disease: a stakeholders' meeting.

Mendell JR, Csimma C, McDonald CM, Escolar DM, Janis S, Porter JD, Hesterlee SE, Howell RR.

Muscle Nerve. 2007 Jan;35(1):8-16.

PMID:
17068768
24.

Fukutin gene mutations in steroid-responsive limb girdle muscular dystrophy.

Godfrey C, Escolar D, Brockington M, Clement EM, Mein R, Jimenez-Mallebrera C, Torelli S, Feng L, Brown SC, Sewry CA, Rutherford M, Shapira Y, Abbs S, Muntoni F.

Ann Neurol. 2006 Nov;60(5):603-10.

PMID:
17044012
25.

Allogeneic stem cell transplantation corrects biochemical derangements in MNGIE.

Hirano M, Martí R, Casali C, Tadesse S, Uldrick T, Fine B, Escolar DM, Valentino ML, Nishino I, Hesdorffer C, Schwartz J, Hawks RG, Martone DL, Cairo MS, DiMauro S, Stanzani M, Garvin JH Jr, Savage DG.

Neurology. 2006 Oct 24;67(8):1458-60. Epub 2006 Sep 13.

26.

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
27.

Translating mighty mice into neuromuscular therapeutics: is bigger muscle better?

Hoffman EP, Escolar D.

Am J Pathol. 2006 Jun;168(6):1775-8. No abstract available.

28.

Nuclear envelope dystrophies show a transcriptional fingerprint suggesting disruption of Rb-MyoD pathways in muscle regeneration.

Bakay M, Wang Z, Melcon G, Schiltz L, Xuan J, Zhao P, Sartorelli V, Seo J, Pegoraro E, Angelini C, Shneiderman B, Escolar D, Chen YW, Winokur ST, Pachman LM, Fan C, Mandler R, Nevo Y, Gordon E, Zhu Y, Dong Y, Wang Y, Hoffman EP.

Brain. 2006 Apr;129(Pt 4):996-1013. Epub 2006 Feb 14.

PMID:
16478798
29.

Update on diagnosis and treatment of hereditary and acquired polyneuropathies in childhood.

Rocha CT, Escolar DM.

Suppl Clin Neurophysiol. 2004;57:255-71. Review. No abstract available.

PMID:
16106624
30.

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
31.

Simulation of the visible spectra for edible virgin oils: potential uses.

Ayuso J, Haro MR, Escolar D.

Appl Spectrosc. 2004 Apr;58(4):474-80.

PMID:
15104819
32.

Genome-wide homozygosity mapping localizes a gene for autosomal recessive non-progressive infantile ataxia to 20q11-q13.

Tranebjaerg L, Teslovich TM, Jones M, Barmada MM, Fagerheim T, Dahl A, Escolar DM, Trent JM, Gillanders EM, Stephan DA.

Hum Genet. 2003 Aug;113(3):293-5. Epub 2003 Jun 17.

PMID:
12811539
33.

PIP2 binding residues of Kir2.1 are common targets of mutations causing Andersen syndrome.

Donaldson MR, Jensen JL, Tristani-Firouzi M, Tawil R, Bendahhou S, Suarez WA, Cobo AM, Poza JJ, Behr E, Wagstaff J, Szepetowski P, Pereira S, Mozaffar T, Escolar DM, Fu YH, Ptácek LJ.

Neurology. 2003 Jun 10;60(11):1811-6.

PMID:
12796536
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.

Worsening of multifocal motor neuropathy during pregnancy.

Chaudhry V, Escolar DM, Cornblath DR.

Neurology. 2002 Jul 9;59(1):139-41.

PMID:
12105326
36.

Pharmacologic and genetic therapy for childhood muscular dystrophies.

Escolar DM, Scacheri CG.

Curr Neurol Neurosci Rep. 2001 Mar;1(2):168-74. Review.

PMID:
11898513
37.

Severe autosomal recessive rippling muscle disease.

Koul RL, Chand RP, Chacko A, Ali M, Brown KM, Bushnarmuth SR, Escolar DM, Stephan DA.

Muscle Nerve. 2001 Nov;24(11):1542-7.

PMID:
11745958
38.

Inflammatory myopathy and hepatitis C in a pediatric patient: role of liver biopsy in evaluating the severity of liver disease.

Mohan P, Chandra RS, Escolar DM, Luban NL.

Hepatology. 2001 Oct;34(4 Pt 1):851-2. No abstract available.

PMID:
11584390
39.

Gentamicin treatment of Duchenne and Becker muscular dystrophy due to nonsense mutations.

Wagner KR, Hamed S, Hadley DW, Gropman AL, Burstein AH, Escolar DM, Hoffman EP, Fischbeck KH.

Ann Neurol. 2001 Jun;49(6):706-11.

PMID:
11409421
40.

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
41.

The mutation of Pro789 to Leu reduces the activity of the fast-twitch skeletal muscle sarco(endo)plasmic reticulum Ca2+ ATPase (SERCA1) and is associated with Brody disease.

Odermatt A, Barton K, Khanna VK, Mathieu J, Escolar D, Kuntzer T, Karpati G, MacLennan DH.

Hum Genet. 2000 May;106(5):482-91.

PMID:
10914677
42.
43.

Measurement of the concentrations of solutions through chromatic systems.

Escolar D, Haro MR, Saucedo A, Ayuso J, Jiménez A, Alvarez JA.

Appl Opt. 1995 Jul 1;34(19):3731-6. doi: 10.1364/AO.34.003731.

PMID:
21052195
44.

I.R. spectra of para-substituted 4-acetamidebutyranilides.

Escolar D, Roldán DM, Braña MF, Castellano JM.

Farmaco Prat. 1977 Feb;32(2):65-82. No abstract available.

PMID:
849776

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