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Items: 1 to 20 of 27

1.

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 Jul 17. pii: S0140-6736(17)31611-2. doi: 10.1016/S0140-6736(17)31611-2. [Epub ahead of print]

PMID:
28728956
2.

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.

3.

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.

4.

Clinical Follow-Up for Duchenne Muscular Dystrophy Newborn Screening: A Proposal.

Kwon JM, Abdel-Hamid HZ, Al-Zaidy SA, Mendell JR, Kennedy A, Kinnett K, Cwik VA, Street N, Bolen J, Day JW, Connolly AM.

Muscle Nerve. 2016 Aug;54(2):186-91. doi: 10.1002/mus.25185. Epub 2016 Jun 13.

5.

Longitudinal effect of eteplirsen versus historical control on ambulation in Duchenne muscular dystrophy.

Mendell JR, Goemans N, Lowes LP, Alfano LN, Berry K, Shao J, Kaye EM, Mercuri E; Eteplirsen Study Group and Telethon Foundation DMD Italian Network.

Ann Neurol. 2016 Feb;79(2):257-71. doi: 10.1002/ana.24555. Epub 2016 Jan 8.

6.

Twenty-year follow-up of newborn screening for patients with muscular dystrophy.

Chung J, Smith AL, Hughes SC, Niizawa G, Abdel-Hamid HZ, Naylor EW, Hughes T, Clemens PR.

Muscle Nerve. 2016 Apr;53(4):570-8. doi: 10.1002/mus.24880. Epub 2015 Sep 10.

PMID:
26260293
7.

Studies on the effect of pollution on Lake Manzala ecosystem in Port-Said, Damietta and Dakahlia Governorates Egypt.

El-Khayat HM, Mahmoud KM, Gaber HS, Abdel-Hamid H, Abu Taleb HM.

J Egypt Soc Parasitol. 2015 Apr;45(1):153-66.

PMID:
26012230
8.

The link between arterial blood pressure and vasogenic edema in pediatric PRES.

Zuccoli G, Fitzgerald RT, Nardone R, Furtado AD, Abdel-Hamid H.

Neuroradiology. 2015 Aug;57(8):865-6. doi: 10.1007/s00234-015-1532-6. Epub 2015 Apr 24. No abstract available.

PMID:
25903431
9.

A rare cause of reversible ophthalmoplegia: tension pneumocephalus with brainstem compression.

Lindner SA, Pollack IF, Abdel-Hamid H, Zuccoli G.

Neuroradiology. 2015 Jul;57(7):761-2. doi: 10.1007/s00234-015-1509-5. Epub 2015 Mar 15. No abstract available.

PMID:
25773200
10.

Guillain Barré syndrome-related posterior reversible encephalopathy syndrome.

Zuccoli G, Nardone R, Hoda AH.

Neuroradiology. 2015 Jul;57(7):755-6. doi: 10.1007/s00234-015-1499-3. Epub 2015 Feb 5. No abstract available.

PMID:
25652259
11.

Biological and hematological responses of Biomphalaria alexandrina to mycobiosynthsis silver nanoparticles.

Abdel-Hamid H, Mekawey AA.

J Egypt Soc Parasitol. 2014 Dec;44(3):627-37.

PMID:
25643504
12.

Molluscicidal and antischistosomal activities of methanol extracts and isolated compounds from Eucalyptus globulus and Melaleuca styphelioides.

Al-Sayed E, Hamid HA, Abu El Einin HM.

Pharm Biol. 2014 Jun;52(6):698-705. doi: 10.3109/13880209.2013.865240. Epub 2014 Feb 7.

PMID:
24824322
13.

Child neurology: tick paralysis: a diagnosis not to miss.

Chagnon SL, Naik M, Abdel-Hamid H.

Neurology. 2014 Mar 18;82(11):e91-3. doi: 10.1212/WNL.0000000000000216.

PMID:
24638220
14.

Spinal Deformity in Bethlem Myopathy.

Dede O, Abdel-Hamid HZ, Deeney VF.

Spine Deform. 2014 Mar;2(2):143-151. doi: 10.1016/j.jspd.2013.11.003. Epub 2014 Mar 5.

PMID:
27927380
15.

Cooperative International Neuromuscular Research Group Duchenne Natural History Study demonstrates insufficient diagnosis and treatment of cardiomyopathy in Duchenne muscular dystrophy.

Spurney C, Shimizu R, Morgenroth LP, Kolski H, Gordish-Dressman H, Clemens PR; CINRG Investigators.

Muscle Nerve. 2014 Aug;50(2):250-6. doi: 10.1002/mus.24163. Epub 2014 May 14.

16.

Parental attitudes toward newborn screening for Duchenne/Becker muscular dystrophy and spinal muscular atrophy.

Wood MF, Hughes SC, Hache LP, Naylor EW, Abdel-Hamid HZ, Barmada MM, Dobrowolski SF, Stickler DE, Clemens PR.

Muscle Nerve. 2014 Jun;49(6):822-8. doi: 10.1002/mus.24100.

PMID:
24307279
17.

Eteplirsen for the treatment of Duchenne muscular dystrophy.

Mendell JR, Rodino-Klapac LR, Sahenk Z, Roush K, Bird L, Lowes LP, Alfano L, Gomez AM, Lewis S, Kota J, Malik V, Shontz K, Walker CM, Flanigan KM, Corridore M, Kean JR, Allen HD, Shilling C, Melia KR, Sazani P, Saoud JB, Kaye EM; Eteplirsen Study Group.

Ann Neurol. 2013 Nov;74(5):637-47. doi: 10.1002/ana.23982. Epub 2013 Sep 10.

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 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.

20.

Child neurology: Pompe disease: new horizons.

Rajan DS, Abdel-Hamid H.

Neurology. 2012 Dec 4;79(23):e197-200. doi: 10.1212/WNL.0b013e318276893c. No abstract available.

PMID:
23212194

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