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Auteur Mah JK |
Documents disponibles écrits par cet auteur



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Multi-Omics Identifies Circulating miRNA and Protein Biomarkers for Facioscapulohumeral Dystrophy
Heier CR, Zhang A, Nguyen NY, et al.
Journal of personalized medicine, 2020, 10, 4, p 236
Revue : Journal of personalized medicine, 10, 4 Titre : Multi-Omics Identifies Circulating miRNA and Protein Biomarkers for Facioscapulohumeral Dystrophy Type de document : Article Auteurs : Heier CR ; Zhang A ; Nguyen NY ; Tully CB ; Panigrahi A ; Gordish-Dressman H ; Pandey SN ; Guglieri M ; Ryan MM ; Clemens PR ; Thangarajh M ; Webster R ; Smith EC ; Connolly AM ; McDonald CM ; Karachunski P ; Tulinius M ; Harper A ; Mah JK ; Fiorillo AA ; Chen YW ; Cooperative International Neuromuscular Research Group Cinrg Investigators Année de publication : 19/11/2020 Pages : p 236 Langues : Anglais (eng) Pubmed / DOI : Pubmed : 33228131 / DOI : 10.3390/jpm10040236
N° Profil MNM : 2020121 En ligne : http://www.ncbi.nlm.nih.gov/pubmed/33228131 Avis des lecteurs Aucun avis, ajoutez le vôtre !
(mauvais) 15 (excellent)
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The CINRG Becker Natural History Study: Baseline Characteristics
Clemens PR, Niizawa G, Feng J, et al.
Muscle & Nerve, 2020
Revue : Muscle & Nerve Titre : The CINRG Becker Natural History Study: Baseline Characteristics Type de document : Article Auteurs : Clemens PR ; Niizawa G ; Feng J ; Florence J ; D'Alessandro AS ; Morgenroth LP ; Gorni K ; Guglieri M ; Connolly A ; Wicklund M ; Bertorini T ; Mah JK ; Thangarajh M ; Smith E ; Kuntz N ; McDonald CM ; Henricson EK ; Upadhyayula S ; Byrne B ; Manousakis G ; Harper A ; Bravver E ; Iannaccone S ; Spurney C ; Cnaan A ; Gordish-Dressman H Editeur : United States Année de publication : 06/2020 Langues : Anglais (eng) Pubmed / DOI : Pubmed : 32564389 / DOI : 10.1002/mus.27011
N° Profil MNM : 2020062 En ligne : http://www.ncbi.nlm.nih.gov/pubmed/32564389 Avis des lecteurs Aucun avis, ajoutez le vôtre !
(mauvais) 15 (excellent)
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Randomized phase 2 trial and open-label extension of domagrozumab in Duchenne muscular dystrophy
Wagner KR, Abdel-Hamid HZ, Mah JK, et al.
Neuromuscular disorders : NMD, 2020, 30, 6, p 492
Revue : Neuromuscular disorders : NMD, 30, 6 Titre : Randomized phase 2 trial and open-label extension of domagrozumab in Duchenne muscular dystrophy Type de document : Article Auteurs : Wagner KR ; Abdel-Hamid HZ ; Mah JK ; Campbell C ; Guglieri M ; Muntoni F ; Takeshima Y ; McDonald CM ; Kostera-Pruszczyk A ; Karachunski P ; Butterfield RJ ; Mercuri E ; Fiorillo C ; Bertini ES ; Tian C ; Statland J ; Sadosky AB ; Purohit VS ; Sherlock SP ; Palmer JP ; Binks M ; Charnas L ; Marraffino S ; Wong BL Editeur : England Année de publication : 06/2020 Pages : p 492 Langues : Anglais (eng) Lien associé : NCT02907619 Pubmed / DOI : Pubmed : 32522498 / DOI : 10.1016/j.nmd.2020.05.002
N° Profil MNM : 2020061 En ligne : http://www.ncbi.nlm.nih.gov/pubmed/32522498 Avis des lecteurs Aucun avis, ajoutez le vôtre !
(mauvais) 15 (excellent)
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A national spinal muscular atrophy registry for real world evidence
Hodgkinson V, Oskoui M, Lounsberry J, et al.
The Canadian journal of neurological sciences. Le journal canadien des sciences neurologiques, 2020
Avis des lecteurs Aucun avis, ajoutez le vôtre !
(mauvais) 15 (excellent)
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Safety, Tolerability, and Efficacy of Viltolarsen in Boys With Duchenne Muscular Dystrophy Amenable to Exon 53 Skipping: A Phase 2 Randomized Clinical Trial
Clemens PR, Rao VK, Connolly AM, et al.
JAMA Neurology, 2020
Revue : JAMA Neurology Titre : Safety, Tolerability, and Efficacy of Viltolarsen in Boys With Duchenne Muscular Dystrophy Amenable to Exon 53 Skipping: A Phase 2 Randomized Clinical Trial Type de document : Article Auteurs : Clemens PR ; Rao VK ; Connolly AM ; Harper AD ; Mah JK ; Smith EC ; McDonald CM ; Zaidman CM ; Morgenroth LP ; Osaki H ; Satou Y ; Yamashita T ; Hoffman EP Année de publication : 05/2020 Langues : Anglais (eng) Lien associé : NCT02740972 Pubmed / DOI : Pubmed : 32453377 / DOI : 10.1001/jamaneurol.2020.1264
N° Profil MNM : 2020053 En ligne : http://www.ncbi.nlm.nih.gov/pubmed/32453377 Avis des lecteurs Aucun avis, ajoutez le vôtre !
(mauvais) 15 (excellent)
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Meta-analyses of ataluren randomized controlled trials in nonsense mutation Duchenne muscular dystrophy
Campbell C, Barohn RJ, Bertini E, et al.
Journal of comparative effectiveness research, 2020
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The Canadian Neuromuscular Disease Registry 2010-2019: A Decade of Facilitating Clinical Research Through a Nationwide, Pan-Neuromuscular Disease Registry
Hodgkinson V, Lounsberry J, M'Dahoma S, et al.
Journal of Neuromuscular Diseases, 2020
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Efficacy and safety of vamorolone in Duchenne muscular dystrophy: An 18-month interim analysis of a non-randomized open-label extension study
Smith EC, Conklin LS, Hoffman EP, et al.
PLoS medicine, 2020, 17, 9
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Disease-specific and glucocorticoid-responsive serum biomarkers for Duchenne Muscular Dystrophy
Hathout Y, Liang C, Ogundele M, et al.
Scientific Reports, 2019, 9, 1
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Vamorolone trial in Duchenne muscular dystrophy shows dose-related improvement of muscle function
Hoffman EP, Schwartz BD, Mengle-Gaw LJ, et al.
Neurology, 2019, 93, 13
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A Pediatric Review of Facioscapulohumeral Muscular Dystrophy
Mah JK, Chen YW
Journal of pediatric neurology : JPN, 2018, 16, 4, p 222
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Current and Emerging Therapies for Duchenne Muscular Dystrophy
Crone M, Crone M, Mah JK
Current treatment options in neurology, 2018, 20, 8
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An Overview of Recent Therapeutics Advances for Duchenne Muscular Dystrophy
Mah JK
Methods in molecular biology, 2018, 1687, p 3
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A multinational study on motor function in early-onset FSHD
Mah JK, Feng J, Jacobs MB, et al.
Neurology, 2018, 90, 15
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The Canadian Neuromuscular Disease Registry: Connecting patients to national and international research opportunities
Wei Y, McCormick A, MacKenzie A, et al.
Paediatrics & child health, 2018, 23, 1, p 20
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Phase IIa trial in Duchenne muscular dystrophy shows vamorolone is a first-in-class dissociative steroidal anti-inflammatory drug
Conklin LS, Conklin LS, Damsker JM, et al.
Pharmacological Research, 2018, 136, p 140
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A checklist for clinical trials in rare disease: obstacles and anticipatory actions-lessons learned from the FOR-DMD trial
Crow RA, Hart KA, McDermott MP, et al.
Trials, 2018, 19, 1
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Cardiac manifestations of congenital LMNA-related muscular dystrophy in children: three case reports and recommendations for care
Heller F, Dabaj I, Mah JK, et al.
Cardiology in the young, 2017, 27, 6, p 1076
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Developing standardized corticosteroid treatment for Duchenne muscular dystrophy
Guglieri M, Bushby K, McDermott MP, et al.
Contemporary clinical trials, 2017, 58, p 34
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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, et al.
Muscle & Nerve, 2017, 55, 4, p 458
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Early onset facioscapulohumeral dystrophy - a systematic review using individual patient data
Goselink RJM, Voermans NC, Okkersen K, et al.
Neuromuscular disorders : NMD, 2017, 27, 12, p 1077
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Facioscapulohumeral dystrophy in children: design of a prospective, observational study on natural history, predictors and clinical impact (iFocus FSHD)
Goselink RJ, Schreuder TH, Mul K, et al.
BMC neurology, 2016, 16, 138
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Clinical practice considerations in facioscapulohumeral muscular dystrophy Sydney, Australia 21 September 2015
Tawil R, Mah JK, Baker S, et al.
Neuromuscular disorders : NMD, 2016, 26, 7, p 462
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Serum pharmacodynamic biomarkers for chronic corticosteroid treatment of children
Hathout Y, Conklin LS, Seol H, et al.
Scientific Reports, 2016, 6
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A Systematic Review and Meta-analysis on the Epidemiology of the Muscular Dystrophies
Mah JK, Korngut L, Fiest KM, et al.
The Canadian journal of neurological sciences. Le journal canadien des sciences neurologiques, 2016, 43, 1, p 163
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