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Items: 1 to 20 of 162

1.

A Rapid Review of eHealth Interventions Addressing the Continuum of HIV Care (2007-2017).

Henny KD, Wilkes AL, McDonald CM, Denson DJ, Neumann MS.

AIDS Behav. 2017 Oct 5. doi: 10.1007/s10461-017-1923-2. [Epub ahead of print]

PMID:
28983684
2.

A phase 3 randomized placebo-controlled trial of tadalafil for Duchenne muscular dystrophy.

Victor RG, Sweeney HL, Finkel R, McDonald CM, Byrne B, Eagle M, Goemans N, Vandenborne K, Dubrovsky AL, Topaloglu H, Miceli MC, Furlong P, Landry J, Elashoff R, Cox D; Tadalafil DMD Study Group.

Neurology. 2017 Oct 24;89(17):1811-1820. doi: 10.1212/WNL.0000000000004570. Epub 2017 Sep 29.

3.

Zinc and multivitamin supplementation have contrasting effects on infant iron status: a randomized, double-blind, placebo-controlled clinical trial.

Carter RC, Kupka R, Manji K, McDonald CM, Aboud S, Erhardt JG, Gosselin K, Kisenge R, Liu E, Fawzi W, Duggan CP.

Eur J Clin Nutr. 2017 Sep 6. doi: 10.1038/ejcn.2017.138. [Epub ahead of print]

PMID:
28876332
4.

Ataluren in patients with nonsense mutation Duchenne muscular dystrophy (ACT DMD): a multicentre, randomised, double-blind, placebo-controlled, phase 3 trial.

McDonald CM, Campbell C, Torricelli RE, Finkel RS, Flanigan KM, Goemans N, Heydemann P, Kaminska A, Kirschner J, Muntoni F, Osorio AN, Schara U, Sejersen T, Shieh PB, Sweeney HL, Topaloglu H, Tulinius M, Vilchez JJ, Voit T, Wong B, Elfring G, Kroger H, Luo X, McIntosh J, Ong T, Riebling P, Souza M, Spiegel RJ, Peltz SW, Mercuri E; Clinical Evaluator Training Group; ACT DMD Study Group.

Lancet. 2017 Sep 23;390(10101):1489-1498. doi: 10.1016/S0140-6736(17)31611-2. Epub 2017 Jul 17.

PMID:
28728956
5.

Longitudinal community walking activity in Duchenne muscular dystrophy.

Fowler EG, Staudt LA, Heberer KR, Sienko SE, Buckon CE, Bagley AM, Sussman MD, McDonald CM.

Muscle Nerve. 2017 Jul 10. doi: 10.1002/mus.25743. [Epub ahead of print]

PMID:
28692198
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.

Presynaptic congenital myasthenic syndrome with a homozygous sequence variant in LAMA5 combines myopia, facial tics, and failure of neuromuscular transmission.

Maselli RA, Arredondo J, Vázquez J, Chong JX; University of Washington Center for Mendelian Genomics, Bamshad MJ, Nickerson DA, Lara M, Ng F, Lo VL, Pytel P, McDonald CM.

Am J Med Genet A. 2017 Aug;173(8):2240-2245. doi: 10.1002/ajmg.a.38291. Epub 2017 May 25.

PMID:
28544784
8.

The Direct Cost of Managing a Rare Disease: Assessing Medical and Pharmacy Costs Associated with Duchenne Muscular Dystrophy in the United States.

Thayer S, Bell C, McDonald CM.

J Manag Care Spec Pharm. 2017 Jun;23(6):633-641. doi: 10.18553/jmcp.2017.23.6.633.

9.

Infant Nutritional Status and Markers of Environmental Enteric Dysfunction are Associated with Midchildhood Anthropometry and Blood Pressure in Tanzania.

Locks LM, Mwiru RS, Mtisi E, Manji KP, McDonald CM, Liu E, Kupka R, Kisenge R, Aboud S, Gosselin K, Gillman M, Gewirtz AT, Fawzi WW, Duggan CP.

J Pediatr. 2017 Aug;187:225-233.e1. doi: 10.1016/j.jpeds.2017.04.005. Epub 2017 May 9.

10.

Developing standardized corticosteroid treatment for Duchenne muscular dystrophy.

Guglieri M, Bushby K, McDermott MP, Hart KA, Tawil R, Martens WB, Herr BE, McColl E, Wilkinson J, Kirschner J, King WM, Eagle M, Brown MW, Willis T, Hirtz D, Shieh PB, Straub V, Childs AM, Ciafaloni E, Butterfield RJ, Horrocks I, Spinty S, Flanigan KM, Kuntz NL, Baranello G, Roper H, Morrison L, Mah JK, Manzur AY, McDonald CM, Schara U, von der Hagen M, Barohn RJ, Campbell C, Darras BT, Finkel RS, Vita G, Hughes I, Mongini T, Pegoraro E, Wicklund M, Wilichowski E, Bryan Burnette W, Howard JF, McMillan HJ, Thangarajh M, Griggs RC.

Contemp Clin Trials. 2017 Jul;58:34-39. doi: 10.1016/j.cct.2017.04.008. Epub 2017 Apr 24.

PMID:
28450193
11.

William M. Fowler, Jr, MD, 1926-2017.

Carter GT, McDonald CM.

PM R. 2017 May;9(5):540-541. doi: 10.1016/j.pmrj.2017.03.003. Epub 2017 Mar 25. No abstract available.

PMID:
28347900
12.

In memoriam: William M. Fowler Jr, MD.

McDonald CM, Carter GT.

Muscle Nerve. 2017 May;55(5):617-618. doi: 10.1002/mus.25640. Epub 2017 Mar 26. No abstract available.

PMID:
28271524
13.

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.

14.

Evidence for ACTN3 as a genetic modifier of Duchenne muscular dystrophy.

Hogarth MW, Houweling PJ, Thomas KC, Gordish-Dressman H, Bello L; Cooperative International Neuromuscular Research Group (CINRG), Pegoraro E, Hoffman EP, Head SI, North KN.

Nat Commun. 2017 Jan 31;8:14143. doi: 10.1038/ncomms14143.

15.

High Burden of Morbidity and Mortality but Not Growth Failure in Infants Exposed to but Uninfected with Human Immunodeficiency Virus in Tanzania.

Locks LM, Manji KP, Kupka R, Liu E, Kisenge R, McDonald CM, Aboud S, Wang M, Fawzi WW, Duggan CP.

J Pediatr. 2017 Jan;180:191-199.e2. doi: 10.1016/j.jpeds.2016.09.040. Epub 2016 Nov 7.

PMID:
27829511
16.

Association Study of Exon Variants in the NF-κB and TGFβ Pathways Identifies CD40 as a Modifier of Duchenne Muscular Dystrophy.

Bello L, Flanigan KM, Weiss RB; United Dystrophinopathy Project, Spitali P, Aartsma-Rus A, Muntoni F, Zaharieva I, Ferlini A, Mercuri E, Tuffery-Giraud S, Claustres M, Straub V, Lochmüller H, Barp A, Vianello S, Pegoraro E, Punetha J, Gordish-Dressman H, Giri M, McDonald CM, Hoffman EP; Cooperative International Neuromuscular Research Group.

Am J Hum Genet. 2016 Nov 3;99(5):1163-1171. doi: 10.1016/j.ajhg.2016.08.023. Epub 2016 Oct 13.

17.

Enteral tube feeding for individuals with cystic fibrosis: Cystic Fibrosis Foundation evidence-informed guidelines.

Schwarzenberg SJ, Hempstead SE, McDonald CM, Powers SW, Wooldridge J, Blair S, Freedman S, Harrington E, Murphy PJ, Palmer L, Schrader AE, Shiel K, Sullivan J, Wallentine M, Marshall BC, Leonard AR.

J Cyst Fibros. 2016 Nov;15(6):724-735. doi: 10.1016/j.jcf.2016.08.004. Epub 2016 Sep 3. Review.

18.

Treatment effect of idebenone on inspiratory function in patients with Duchenne muscular dystrophy.

Buyse GM, Voit T, Schara U, Straathof CS, D'Angelo MG, Bernert G, Cuisset JM, Finkel RS, Goemans N, Rummey C, Leinonen M, Mayer OH, Spagnolo P, Meier T, McDonald CM; DELOS Study Group.

Pediatr Pulmonol. 2017 Apr;52(4):508-515. doi: 10.1002/ppul.23547. Epub 2016 Aug 29.

19.

Prednisone and Deflazacort in Duchenne Muscular Dystrophy: Do They Play a Different Role in Child Behavior and Perceived Quality of Life?

Sienko S, Buckon C, Fowler E, Bagley A, Staudt L, Sison-Williamson M, Zebracki K, McDonald CM, Sussman M.

PLoS Curr. 2016 Jun 17;8. pii: ecurrents.md.7628d9c014bfa29f821a5cd19723bbaa. doi: 10.1371/currents.md.7628d9c014bfa29f821a5cd19723bbaa.

20.

Can Quantitative Muscle Strength and Functional Motor Ability Differentiate the Influence of Age and Corticosteroids in Ambulatory Boys with Duchenne Muscular Dystrophy?

Buckon C, Sienko S, Bagley A, Sison-Williamson M, Fowler E, Staudt L, Heberer K, McDonald CM, Sussman M.

PLoS Curr. 2016 Jul 8;8. pii: ecurrents.md.1ced64dff945f8958221fddcd4ee60b0. doi: 10.1371/currents.md.1ced64dff945f8958221fddcd4ee60b0.

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