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Volumn 2, Issue , 2015, Pages S83-S87

Looking Forward to New Therapies: A Personal Perspective on the Translational Landscape for Muscular Dystrophies

(1)  Bushby, Kate a  

a NONE

Author keywords

[No Author keywords available]

Indexed keywords

FUNDING; HEALTH CARE DELIVERY; HEALTH SERVICE; HUMAN; MUSCULAR DYSTROPHY; NONHUMAN; OUTCOME ASSESSMENT; PRIORITY JOURNAL; RARE DISEASE; REVIEW; THERAPY; TRANSLATIONAL RESEARCH;

EID: 84964569872     PISSN: 22143599     EISSN: 22143602     Source Type: Journal    
DOI: 10.3233/JND-150096     Document Type: Review
Times cited : (1)

References (25)
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  • 2
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    • Bushby, K.1    Lochmuller, H.2    Lynn, S.3    Straub, V.4
  • 3
    • 84902180595 scopus 로고    scopus 로고
    • Expanding rare disease drug trials based on shared molecular etiology
    • Brooks PJ, Tagle DA, Groft S. Expanding rare disease drug trials based on shared molecular etiology. Nat Biotechnol. 2014;32:515-8.
    • (2014) Nat Biotechnol. , vol.32 , pp. 515-518
    • Brooks, P.J.1    Tagle, D.A.2    Groft, S.3
  • 4
    • 84887405291 scopus 로고    scopus 로고
    • Billions spent on rare diseases
    • Rohn J. Billions spent on rare diseases. Nat Biotechnol. 2013;31:368.
    • (2013) Nat Biotechnol. , vol.31 , pp. 368
    • Rohn, J.1
  • 8
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    • Choosing the right clinical outcome measure: From the patient to the statistician and back
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    • (2011) Neuromuscular Disorders: NMD , vol.21 , pp. 16-19
    • Mercuri, E.1    Mazzone, E.2
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    • Developing standard procedures for pre-clinical efficacy studies in mouse models of spinal muscular atrophy: Report of the expert workshop "pre-clinical testing for SMA" Zurich, March 29-30th 2010
    • Willmann R, Dubach J, Chen K, Network T-NN. Developing standard procedures for pre-clinical efficacy studies in mouse models of spinal muscular atrophy: Report of the expert workshop "Pre-clinical testing for SMA", Zurich, March 29-30th 2010. Neuromuscular Disorders: NMD 2011;21: 74-7.
    • (2011) Neuromuscular Disorders: NMD , vol.21 , pp. 74-77
    • Willmann, R.1    Dubach, J.2    Chen, K.3    Network, T.-N.N.4
  • 11
    • 67649672101 scopus 로고    scopus 로고
    • Developing standard procedures for murine and canine efficacy studies of DMD therapeutics: Report of two expert workshops on "pre-clinical testing for Duchenne dystrophy" Washington DC, October 27th-28th 2007 and Zurich, June 30th-July 1st 2008
    • The Wellstone Muscular Dystrophy Cooperative Research N
    • Nagaraju K, Willmann R, Network T-N, The Wellstone Muscular Dystrophy Cooperative Research N. Developing standard procedures for murine and canine efficacy studies of DMD therapeutics: Report of two expert workshops on "Pre-clinical testing for Duchenne dystrophy". Washington DC, October 27th-28th 2007 and Zurich, June 30th-July 1st 2008. Neuromuscular Disorders: NMD. 2009;19:502-6.
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    • Nagaraju, K.1    Willmann, R.2    Network, T.-N.3
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    • Bushby K, Connor E. Clinical outcome measures for trials in Duchenne muscular dystrophy: Report from International Working Group meetings. J Clin Invest. 2011;1:1217-35.
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    • Bushby, K.1    Connor, E.2
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    • TREATNMD workshop on outcome measures in experimental trials for DMD, 30th June-1st July 2007, Naarden, The Netherlands; conjoint Institute of Myology TREAT-NMD meeting on physical activity monitoring in neuromuscular disorders, 11th July 2007, Paris, France
    • Mercuri E, Mayhew A, Muntoni F, Messina S, Straub V, Van Ommen GJ, Voit T, Bertini E, Bushby K. Towards harmonisation of outcome measures for DMD and SMA within TREAT-NMD; report of three expert workshops: TREAT-NMD/ENMC workshop on outcome measures, 12th-13th May 2007, Naarden, The Netherlands; TREATNMD workshop on outcome measures in experimental trials for DMD, 30th June-1st July 2007, Naarden, The Netherlands; conjoint Institute of Myology TREAT-NMD meeting on physical activity monitoring in neuromuscular disorders, 11th July 2007, Paris, France. Neuromuscular Disorders: NMD. 2008;18:894-903.
    • (2008) Neuromuscular Disorders: NMD. , vol.18 , pp. 894-903
    • Mercuri, E.1    Mayhew, A.2    Muntoni, F.3    Messina, S.4    Straub, V.5    Van Ommen, G.J.6    Voit, T.7    Bertini, E.8    Bushby, K.9


* 이 정보는 Elsevier사의 SCOPUS DB에서 KISTI가 분석하여 추출한 것입니다.