-
2
-
-
0001095388
-
Clinical forum: treatment efficacy. The notion of clinically significant change
-
Bain BA, Dollaghan CA. Clinical forum: treatment efficacy. The notion of clinically significant change. Language, Speech, and Hearing Services in Schools 1991; 22(10): 264-270.
-
(1991)
Language, Speech, and Hearing Services in Schools
, vol.22
, Issue.10
, pp. 264-270
-
-
Bain, B.A.1
Dollaghan, C.A.2
-
3
-
-
33645769504
-
A motor function measurement scale for neuromuscular diseases-description and validation study (in French)
-
Groupe d'Etude MFM.
-
Bérard C, Payan C, Fermanian J, Girardot F, Groupe d'Etude MFM. A motor function measurement scale for neuromuscular diseases-description and validation study (in French). Revue Neurologique (Paris) 2006; 162(4): 485-493.
-
(2006)
Revue Neurologique (Paris)
, vol.162
, Issue.4
, pp. 485-493
-
-
Bérard, C.1
Payan, C.2
Fermanian, J.3
Girardot, F.4
-
4
-
-
21244460238
-
A motor function measure for neuromuscular diseases. Construction and validation study
-
MFM Collaborative Study Group.
-
Bérard C, Payan C, Hodgkinson I, Fermanian J, MFM Collaborative Study Group. A motor function measure for neuromuscular diseases. Construction and validation study. Neuromuscular Disorders 2005; 15(7): 463-470.
-
(2005)
Neuromuscular Disorders
, vol.15
, Issue.7
, pp. 463-470
-
-
Bérard, C.1
Payan, C.2
Hodgkinson, I.3
Fermanian, J.4
-
5
-
-
0019522162
-
Clinical trial in Duchenne dystrophy. I. The design of the protocol
-
Brooke MH, Griggs RC, Mendell JR, Fenichel GM, Shumate JB, Pellegrino RJ. Clinical trial in Duchenne dystrophy. I. The design of the protocol. Muscle & Nerve 1981; 4(3): 186-197.
-
(1981)
Muscle & Nerve
, vol.4
, Issue.3
, pp. 186-197
-
-
Brooke, M.H.1
Griggs, R.C.2
Mendell, J.R.3
Fenichel, G.M.4
Shumate, J.B.5
Pellegrino, R.J.6
-
6
-
-
84862016649
-
Current progress with the systemic administration trial of AVI-4658, a novel phosphorodiamidate morpholino oligomer (PMO) skipping exon 51 in Duchenne muscular dystrophy (DMD). World Muscle Society Meeting, Geneva, 9-12 September 2009
-
Cirak S, Muntoni F, Guglieri M, Bushby K, Leow S, Shrewsbury S. Current progress with the systemic administration trial of AVI-4658, a novel phosphorodiamidate morpholino oligomer (PMO) skipping exon 51 in Duchenne muscular dystrophy (DMD). World Muscle Society Meeting, Geneva, 9-12 September 2009. Neuromuscular Disorders 2009; 19(8-9): 511-674.
-
(2009)
Neuromuscular Disorders
, vol.19
, Issue.8-9
, pp. 511-674
-
-
Cirak, S.1
Muntoni, F.2
Guglieri, M.3
Bushby, K.4
Leow, S.5
Shrewsbury, S.6
-
7
-
-
77949442788
-
The PedsQL in pediatric patients with Duchenne muscular dystrophy: feasibility, reliability, and validity of the Pediatric Quality of Life Inventory Neuromuscular Module and Generic Core Scales
-
Davis SE, Hynan LS, Limbers CA, Andersen CM, Greene MC, Varni JW, Iannaccone ST. The PedsQL in pediatric patients with Duchenne muscular dystrophy: feasibility, reliability, and validity of the Pediatric Quality of Life Inventory Neuromuscular Module and Generic Core Scales. Journal of Clinical Neuromuscular Disease 2010; 11(3): 97-109.
-
(2010)
Journal of Clinical Neuromuscular Disease
, vol.11
, Issue.3
, pp. 97-109
-
-
Davis, S.E.1
Hynan, L.S.2
Limbers, C.A.3
Andersen, C.M.4
Greene, M.C.5
Varni, J.W.6
Iannaccone, S.T.7
-
8
-
-
0004292396
-
-
Emery AEH, Muntoni F (eds), 3rd edition). Oxford: Oxford University Press
-
Emery EH, Muntoni F. In: Emery AEH, Muntoni F (eds), Duchenne Muscular Dystrophy (3rd edition). Oxford: Oxford University Press, 2003.
-
(2003)
Duchenne Muscular Dystrophy
-
-
Emery, E.H.1
Muntoni, F.2
-
9
-
-
84965886444
-
The equivalence of weighted kappa and the intraclass correlation coefficient as measures of reliability
-
Fleiss JL, Cohen J. The equivalence of weighted kappa and the intraclass correlation coefficient as measures of reliability. Educational and Psychological Measurement 1973; 33: 613-619.
-
(1973)
Educational and Psychological Measurement
, vol.33
, pp. 613-619
-
-
Fleiss, J.L.1
Cohen, J.2
-
10
-
-
69949107887
-
Local restoration of dystrophin expression in Duchenne muscular dystrophy: a single blind, placebo-controlled dose escalation study using morpholino antisense oligomer AVI-4658
-
Kinali M, Arechavala-Gomeza V, Feng L, Cirak S, Hunt D, Adkin C, Guglieri M, Ashton E, Abbs S, Nihoyannopoulos P, Garralda ME, Rutherford M, McCulley C, Popplewell L, Graham IR, Dickson G, Wood MJ, Wells DJ, Wilton SD, Kole R, Straub V, Bushby K, Sewry C, Morgan JE, Muntoni F. Local restoration of dystrophin expression in Duchenne muscular dystrophy: a single blind, placebo-controlled dose escalation study using morpholino antisense oligomer AVI-4658. Lancet Neurology 2009; 8(10): 918-928.
-
(2009)
Lancet Neurology
, vol.8
, Issue.10
, pp. 918-928
-
-
Kinali, M.1
Arechavala-Gomeza, V.2
Feng, L.3
Cirak, S.4
Hunt, D.5
Adkin, C.6
Guglieri, M.7
Ashton, E.8
Abbs, S.9
Nihoyannopoulos, P.10
Garralda, M.E.11
Rutherford, M.12
McCulley, C.13
Popplewell, L.14
Graham, I.R.15
Dickson, G.16
Wood, M.J.17
Wells, D.J.18
Wilton, S.D.19
Kole, R.20
Straub, V.21
Bushby, K.22
Sewry, C.23
Morgan, J.E.24
Muntoni, F.25
more..
-
11
-
-
26944476254
-
Quality of life, physical disability, and respiratory impairment in Duchenne muscular dystrophy
-
Kohler M, Clarenbach CF, Böni L, Brack T, Russi EW, Bloch KE. Quality of life, physical disability, and respiratory impairment in Duchenne muscular dystrophy. American Journal of Respiratory and Critical Care Medicine 2005; 172(8): 1032-1036.
-
(2005)
American Journal of Respiratory and Critical Care Medicine
, vol.172
, Issue.8
, pp. 1032-1036
-
-
Kohler, M.1
Clarenbach, C.F.2
Böni, L.3
Brack, T.4
Russi, E.W.5
Bloch, K.E.6
-
12
-
-
41749096186
-
Glucocorticoid corticosteroids for Duchenne muscular dystrophy
-
Issue 1. Art. No. CD003725. DOI: 10.1002/14651858.CD003725.pub3
-
Manzur AY, Kuntzer T, Pike M, Swan AV. Glucocorticoid corticosteroids for Duchenne muscular dystrophy. Cochrane Database of Systematic Reviews 2008a, Issue 1. Art. No. CD003725. DOI: 10.1002/14651858.CD003725.pub3.
-
(2008)
Cochrane Database of Systematic Reviews
-
-
Manzur, A.Y.1
Kuntzer, T.2
Pike, M.3
Swan, A.V.4
-
13
-
-
55349084415
-
Update on the management of Duchenne muscular dystrophy
-
Invited review.
-
Manzur AY, Kinali M, Muntoni F. Update on the management of Duchenne muscular dystrophy. Archives of Disease in Childhood 2008b; 93(11): 986-990. Invited review.
-
(2008)
Archives of Disease in Childhood
, vol.93
, Issue.11
, pp. 986-990
-
-
Manzur, A.Y.1
Kinali, M.2
Muntoni, F.3
-
14
-
-
33845989867
-
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. Reliable surrogate outcome measures in multicenter clinical trials of Duchenne muscular dystrophy. Muscle & Nerve 2007; 35(1): 36-42.
-
(2007)
Muscle & Nerve
, vol.35
, Issue.1
, pp. 36-42
-
-
Mayhew, J.E.1
Florence, J.M.2
Mayhew, T.P.3
Henricson, E.K.4
Leshner, R.T.5
McCarter, R.J.6
Escolar, D.M.7
-
15
-
-
67649842377
-
Reliability of the North Star Ambulatory Assessment in a multicentric setting
-
Mazzone ES, Messina S, Vasco G, Main M, Eagle M, D'Amico A, Doglio L, Politano L, Cavallaro F, Frosini S, Bello L, Magri F, Corlatti A, Zucchini E, Brancalion B, Rossi F, Ferretti M, Motta MG, Cecio MR, Berardinelli A, Alfieri P, Mongini T, Pini A, Astrea G, Battini R, Comi G, Pegoraro E, Morandi L, Pane M, Angelini C, Bruno C, Villanova M, Vita G, Donati MA, Bertini E, Mercuri E. Reliability of the North Star Ambulatory Assessment in a multicentric setting. Neuromuscular Disorders 2009; 19(7): 458-461.
-
(2009)
Neuromuscular Disorders
, vol.19
, Issue.7
, pp. 458-461
-
-
Mazzone, E.S.1
Messina, S.2
Vasco, G.3
Main, M.4
Eagle, M.5
D'Amico, A.6
Doglio, L.7
Politano, L.8
Cavallaro, F.9
Frosini, S.10
Bello, L.11
Magri, F.12
Corlatti, A.13
Zucchini, E.14
Brancalion, B.15
Rossi, F.16
Ferretti, M.17
Motta, M.G.18
Cecio, M.R.19
Berardinelli, A.20
Alfieri, P.21
Mongini, T.22
Pini, A.23
Astrea, G.24
Battini, R.25
Comi, G.26
Pegoraro, E.27
Morandi, L.28
Pane, M.29
Angelini, C.30
Bruno, C.31
Villanova, M.32
Vita, G.33
Donati, M.A.34
Bertini, E.35
Mercuri, E.36
more..
-
16
-
-
77950210043
-
The 6-minute walk test as a new outcome measure in Duchenne muscular dystrophy
-
McDonald CM, Henricson EK, Han JJ, Abresch RT, Nicorici A, Elfring GL, Atkinson L, Reha A, Hirawat S, Miller LL. The 6-minute walk test as a new outcome measure in Duchenne muscular dystrophy. Muscle & Nerve 2010; 41(4): 500-510.
-
(2010)
Muscle & Nerve
, vol.41
, Issue.4
, pp. 500-510
-
-
McDonald, C.M.1
Henricson, E.K.2
Han, J.J.3
Abresch, R.T.4
Nicorici, A.5
Elfring, G.L.6
Atkinson, L.7
Reha, A.8
Hirawat, S.9
Miller, L.L.10
-
17
-
-
0036838246
-
Physical activity, health impairments, and disability in neuromuscular disease
-
McDonald CM. Physical activity, health impairments, and disability in neuromuscular disease. American Journal of Physical Medicine & Rehabilitation 2002; 81(11 Suppl): S108-S120.
-
(2002)
American Journal of Physical Medicine & Rehabilitation
, vol.81
, Issue.11 SUPPL.
-
-
McDonald, C.M.1
-
18
-
-
53249098299
-
-
TREAT-NMD Neuromuscular Network. 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; TREAT-NMD workshop on outcome measures in experimental trials for DMD, 30th June-1st July, 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. TREAT-NMD Neuromuscular Network. 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; TREAT-NMD workshop on outcome measures in experimental trials for DMD, 30th June-1st July, Naarden, The Netherlands; Conjoint Institute of Myology TREAT-NMD meeting on physical activity monitoring in neuromuscular disorders, 11th July 2007, Paris, France. Neuromuscular Disorders 2008; 18: 894-903.
-
(2008)
Neuromuscular Disorders
, 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
-
20
-
-
33646672126
-
Measurement in Duchenne muscular dystrophy: considerations in the development of a neuromuscular assessment tool
-
Scott E, Mawson SJ. Measurement in Duchenne muscular dystrophy: considerations in the development of a neuromuscular assessment tool. Developmental Medicine and Child Neurology 2006; 48(6): 540-544.
-
(2006)
Developmental Medicine and Child Neurology
, vol.48
, Issue.6
, pp. 540-544
-
-
Scott, E.1
Mawson, S.J.2
-
21
-
-
84862001275
-
NorthStar and SMARTnet: development of paediatric neuromuscular disease networks in the UK
-
No. 108
-
Scott E, Mayhew A, Muntoni F, Manzur AY. NorthStar and SMARTnet: development of paediatric neuromuscular disease networks in the UK. Developmental Medicine and Child Neurology 2007; 49(Supplement No. 108): 29.
-
(2007)
Developmental Medicine and Child Neurology
, vol.49
, Issue.SUPPLEMENT
, pp. 29
-
-
Scott, E.1
Mayhew, A.2
Muntoni, F.3
Manzur, A.Y.4
-
22
-
-
0019948028
-
Quantitation of muscle function in children: a prospective study in Duchenne muscular dystrophy
-
Scott OM, Hyde SA, Goddard C, Dubowitz, V. Quantitation of muscle function in children: a prospective study in Duchenne muscular dystrophy. Muscle & Nerve 1982; 5(4): 291-301.
-
(1982)
Muscle & Nerve
, vol.5
, Issue.4
, pp. 291-301
-
-
Scott, O.M.1
Hyde, S.A.2
Goddard, C.3
Dubowitz, V.4
-
23
-
-
0041841956
-
Validity of the EK Scale: a functional assessment of non-ambulatory individuals with Duchenne muscular dystrophy or spinal muscular atrophy
-
Steffensen B, Hyde S, Lyager S, Mattsson E. Validity of the EK Scale: a functional assessment of non-ambulatory individuals with Duchenne muscular dystrophy or spinal muscular atrophy. Physiotherapy Research International 2001; 6(3): 119-134.
-
(2001)
Physiotherapy Research International
, vol.6
, Issue.3
, pp. 119-134
-
-
Steffensen, B.1
Hyde, S.2
Lyager, S.3
Mattsson, E.4
-
24
-
-
0036726945
-
Physical capacity in non-ambulatory people with Duchenne muscular dystrophy or spinal muscular atrophy: a longitudinal study
-
Steffensen BF, Lyager S, Werge B, Rahbek J, Mattson E. Physical capacity in non-ambulatory people with Duchenne muscular dystrophy or spinal muscular atrophy: a longitudinal study. Developmental Medicine and Child Neurology 2002; 44(9): 623-632.
-
(2002)
Developmental Medicine and Child Neurology
, vol.44
, Issue.9
, pp. 623-632
-
-
Steffensen, B.F.1
Lyager, S.2
Werge, B.3
Rahbek, J.4
Mattson, E.5
-
26
-
-
73649200203
-
Management of progressive muscular dystrophy in childhood
-
Vignos PJ, Jr, Spencer GE, Jr, Archibald KC. Management of progressive muscular dystrophy in childhood. JAMA 1963; 184: 89-96.
-
(1963)
JAMA
, vol.184
, pp. 89-96
-
-
Vignos Jr., P.J.1
Spencer Jr., G.E.2
Archibald, K.C.3
|