-
1
-
-
10744229981
-
Phenylbutyrate increases SMN expression in vitro: relevance for treatment of spinal muscular atrophy
-
Andreassi C., Angelozzi C., Tiziano F.D., et al. Phenylbutyrate increases SMN expression in vitro: relevance for treatment of spinal muscular atrophy. Eur J Hum Genet 12 1 (2004) 59-65
-
(2004)
Eur J Hum Genet
, vol.12
, Issue.1
, pp. 59-65
-
-
Andreassi, C.1
Angelozzi, C.2
Tiziano, F.D.3
-
2
-
-
13544258982
-
Phenylbutyrate increases SMN gene expression in spinal muscular atrophy patients
-
Brahe C., Vitali T., Tiziano F.D., et al. Phenylbutyrate increases SMN gene expression in spinal muscular atrophy patients. Eur J Hum Genet 13 2 (2005) 256-259
-
(2005)
Eur J Hum Genet
, vol.13
, Issue.2
, pp. 256-259
-
-
Brahe, C.1
Vitali, T.2
Tiziano, F.D.3
-
3
-
-
4344647889
-
Concerns about the design of clinical trials for spinal muscular atrophy
-
Crawford T.O. Concerns about the design of clinical trials for spinal muscular atrophy. Neuromuscul Disord 14 8-9 (2004) 456-460
-
(2004)
Neuromuscul Disord
, vol.14
, Issue.8-9
, pp. 456-460
-
-
Crawford, T.O.1
-
4
-
-
0034980532
-
Reference values of maximum isometric muscle force obtained in 270 children aged 4-16 years by hand-held dynamometry
-
Beenakker E.A., van der Hoeven J.H., Fock J.M., and Maurits N.M. Reference values of maximum isometric muscle force obtained in 270 children aged 4-16 years by hand-held dynamometry. Neuromuscul Disord 11 5 (2001) 441-446
-
(2001)
Neuromuscul Disord
, vol.11
, Issue.5
, pp. 441-446
-
-
Beenakker, E.A.1
van der Hoeven, J.H.2
Fock, J.M.3
Maurits, N.M.4
-
5
-
-
0042383467
-
Role of gabapentin in spinal muscular atrophy: results of a multicenter, randomized Italian study
-
Merlini L., Solari A., Vita G., et al. Role of gabapentin in spinal muscular atrophy: results of a multicenter, randomized Italian study. J Child Neurol 18 8 (2003) 537-541
-
(2003)
J Child Neurol
, vol.18
, Issue.8
, pp. 537-541
-
-
Merlini, L.1
Solari, A.2
Vita, G.3
-
6
-
-
0035886619
-
A placebo-controlled trial of gabapentin in spinal muscular atrophy
-
Miller R.G., Moore D.H., Dronsky V., et al. A placebo-controlled trial of gabapentin in spinal muscular atrophy. J Neurol Sci 191 1-2 (2001) 127-131
-
(2001)
J Neurol Sci
, vol.191
, Issue.1-2
, pp. 127-131
-
-
Miller, R.G.1
Moore, D.H.2
Dronsky, V.3
-
7
-
-
0036307964
-
Reliability of hand-held dynamometry in spinal muscular atrophy
-
Merlini L., Mazzone E.S., Solari A., and Morandi L. Reliability of hand-held dynamometry in spinal muscular atrophy. Muscle Nerve 26 1 (2002) 64-70
-
(2002)
Muscle Nerve
, vol.26
, Issue.1
, pp. 64-70
-
-
Merlini, L.1
Mazzone, E.S.2
Solari, A.3
Morandi, L.4
-
8
-
-
1842529578
-
Motor function-muscle strength relationship in spinal muscular atrophy
-
Merlini L., Bertini E., Minetti C., et al. Motor function-muscle strength relationship in spinal muscular atrophy. Muscle Nerve 29 4 (2004) 548-552
-
(2004)
Muscle Nerve
, vol.29
, Issue.4
, pp. 548-552
-
-
Merlini, L.1
Bertini, E.2
Minetti, C.3
-
9
-
-
0036362042
-
Review of the reliability and validity of myometry with children
-
Sloan C. Review of the reliability and validity of myometry with children. Phys Occup Ther Pediatr 22 2 (2002) 79-93
-
(2002)
Phys Occup Ther Pediatr
, vol.22
, Issue.2
, pp. 79-93
-
-
Sloan, C.1
-
10
-
-
0043092414
-
The Hammersmith functional motor scale for children with spinal muscular atrophy: a scale to test ability and monitor progress in children with limited ambulation
-
Main M., Kairon H., Mercuri E., and Muntoni F. The Hammersmith functional motor scale for children with spinal muscular atrophy: a scale to test ability and monitor progress in children with limited ambulation. Eur J Paediatr Neurol 7 4 (2003) 155-159
-
(2003)
Eur J Paediatr Neurol
, vol.7
, Issue.4
, pp. 155-159
-
-
Main, M.1
Kairon, H.2
Mercuri, E.3
Muntoni, F.4
-
11
-
-
16644394038
-
Spinal muscular atrophy: survival pattern and functional status
-
Chung B.H., Wong V.C., and Ip P. Spinal muscular atrophy: survival pattern and functional status. Pediatrics 114 5 (2004) e548-e553
-
(2004)
Pediatrics
, vol.114
, Issue.5
-
-
Chung, B.H.1
Wong, V.C.2
Ip, P.3
-
12
-
-
0036717349
-
Outcome measures for pediatric spinal muscular atrophy
-
Iannaccone S.T. Outcome measures for pediatric spinal muscular atrophy. Arch Neurol 59 9 (2002) 1445-1450
-
(2002)
Arch Neurol
, vol.59
, Issue.9
, pp. 1445-1450
-
-
Iannaccone, S.T.1
-
13
-
-
0035044661
-
The predictive value of achieved motor milestones assessed in 441 patients with infantile spinal muscular atrophy types II and III
-
Rudnik-Schoneborn S., Hausmanowa-Petrusewicz I., Borkowska J., and Zerres K. The predictive value of achieved motor milestones assessed in 441 patients with infantile spinal muscular atrophy types II and III. Eur Neurol 45 3 (2001) 174-181
-
(2001)
Eur Neurol
, vol.45
, Issue.3
, pp. 174-181
-
-
Rudnik-Schoneborn, S.1
Hausmanowa-Petrusewicz, I.2
Borkowska, J.3
Zerres, K.4
-
14
-
-
10244245060
-
Function changes in spinal muscular atrophy II and III. The DCN/SMA group
-
Russman B.S., Buncher C.R., White M., Samaha F.J., and Iannaccone S.T. Function changes in spinal muscular atrophy II and III. The DCN/SMA group. Neurology 47 4 (1996) 973-976
-
(1996)
Neurology
, vol.47
, Issue.4
, pp. 973-976
-
-
Russman, B.S.1
Buncher, C.R.2
White, M.3
Samaha, F.J.4
Iannaccone, S.T.5
-
17
-
-
0019522162
-
Clinical trial in Duchenne dystrophy. I. The design of the protocol
-
Brooke M.H., Griggs R.C., Mendell J.R., Fenichel G.M., Shumate J.B., and Pellegrino R.J. Clinical trial in Duchenne dystrophy. I. The design of the protocol. Muscle Nerve 4 3 (1981) 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
-
18
-
-
0019746298
-
The natural history of Duchenne muscular dystrophy: a caveat for therapeutic trials
-
Brooke M.H., Griggs R.C., Mendell J.R., Fenichel G.M., and Shumate J.B. The natural history of Duchenne muscular dystrophy: a caveat for therapeutic trials. Trans Am Neurol Assoc 106 (1981) 195-199
-
(1981)
Trans Am Neurol Assoc
, vol.106
, pp. 195-199
-
-
Brooke, M.H.1
Griggs, R.C.2
Mendell, J.R.3
Fenichel, G.M.4
Shumate, J.B.5
-
19
-
-
0019948028
-
Quantitation of muscle function in children: a prospective study in Duchenne muscular dystrophy
-
Scott O.M., Hyde S.A., Goddard C., and Dubowitz V. Quantitation of muscle function in children: a prospective study in Duchenne muscular dystrophy. Muscle Nerve 5 4 (1982) 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
-
20
-
-
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., and Mattsson E. Validity of the EK scale: a functional assessment of non-ambulatory individuals with Duchenne muscular dystrophy or spinal muscular atrophy. Physiother Res Int 6 3 (2001) 119-134
-
(2001)
Physiother Res Int
, vol.6
, Issue.3
, pp. 119-134
-
-
Steffensen, B.1
Hyde, S.2
Lyager, S.3
Mattsson, E.4
-
21
-
-
0032692481
-
The ALSFRS-R: a revised ALS functional rating scale that incorporates assessments of respiratory function. BDNF ALS study group (phase III)
-
Cedarbaum J.M., Stambler N., Malta E., et al. The ALSFRS-R: a revised ALS functional rating scale that incorporates assessments of respiratory function. BDNF ALS study group (phase III). J Neurol Sci 169 1-2 (1999) 13-21
-
(1999)
J Neurol Sci
, vol.169
, Issue.1-2
, pp. 13-21
-
-
Cedarbaum, J.M.1
Stambler, N.2
Malta, E.3
-
22
-
-
0030678474
-
Performance of the amyotrophic lateral sclerosis functional rating scale (ALSFRS) in multicenter clinical trials
-
Cedarbaum J.M., and Stambler N. Performance of the amyotrophic lateral sclerosis functional rating scale (ALSFRS) in multicenter clinical trials. J Neurol Sci 152 Suppl 1 (1997) S1-S9
-
(1997)
J Neurol Sci
, vol.152
, Issue.SUPPL. 1
-
-
Cedarbaum, J.M.1
Stambler, N.2
-
23
-
-
0023389864
-
Clinical investigation of Duchenne muscular dystrophy. Interesting results in a trial of prednisone
-
Brooke M.H., Fenichel G.M., Griggs R.C., et al. Clinical investigation of Duchenne muscular dystrophy. Interesting results in a trial of prednisone. Arch Neurol 44 8 (1987) 812-817
-
(1987)
Arch Neurol
, vol.44
, Issue.8
, pp. 812-817
-
-
Brooke, M.H.1
Fenichel, G.M.2
Griggs, R.C.3
-
24
-
-
0036893708
-
High dose weekly oral prednisone improves strength in boys with Duchenne muscular dystrophy
-
Connolly A.M., Schierbecker J., Renna R., and Florence J. High dose weekly oral prednisone improves strength in boys with Duchenne muscular dystrophy. Neuromuscul Disord 12 10 (2002) 917-925
-
(2002)
Neuromuscul Disord
, vol.12
, Issue.10
, pp. 917-925
-
-
Connolly, A.M.1
Schierbecker, J.2
Renna, R.3
Florence, J.4
-
25
-
-
0026344234
-
Long-term benefit from prednisone therapy in Duchenne muscular dystrophy
-
Fenichel G.M., Florence J.M., Pestronk A., et al. Long-term benefit from prednisone therapy in Duchenne muscular dystrophy. Neurology 41 12 (1991) 1874-1877
-
(1991)
Neurology
, vol.41
, Issue.12
, pp. 1874-1877
-
-
Fenichel, G.M.1
Florence, J.M.2
Pestronk, A.3
-
26
-
-
0035942323
-
A randomized efficacy and safety trial of oxandrolone in the treatment of Duchenne dystrophy
-
Fenichel G.M., Griggs R.C., Kissel J., et al. A randomized efficacy and safety trial of oxandrolone in the treatment of Duchenne dystrophy. Neurology 56 8 (2001) 1075-1079
-
(2001)
Neurology
, vol.56
, Issue.8
, pp. 1075-1079
-
-
Fenichel, G.M.1
Griggs, R.C.2
Kissel, J.3
-
27
-
-
18244407748
-
Natural history of denervation in SMA: relation to age, SMN2 copy number, and function
-
Swoboda K.J., Prior T.W., Scott C.B., et al. Natural history of denervation in SMA: relation to age, SMN2 copy number, and function. Ann Neurol 57 5 (2005) 704-712
-
(2005)
Ann Neurol
, vol.57
, Issue.5
, pp. 704-712
-
-
Swoboda, K.J.1
Prior, T.W.2
Scott, C.B.3
-
28
-
-
0027070895
-
Construction and validation of the Alberta infant motor scale (AIMS)
-
Piper M.C., Pinnell L.E., Darrah J., Maguire T., and Byrne P.J. Construction and validation of the Alberta infant motor scale (AIMS). Can J Public Health 83 Suppl 2 (1992) S46-S50
-
(1992)
Can J Public Health
, vol.83
, Issue.SUPPL. 2
-
-
Piper, M.C.1
Pinnell, L.E.2
Darrah, J.3
Maguire, T.4
Byrne, P.J.5
-
29
-
-
84914069824
-
-
Spastic International Medical Publications, London
-
Egan D.F., Illingworth R.S., and Mac Keith R.C. Developmental screening 0-5 years, clinics in developmental medicine (1969), Spastic International Medical Publications, London
-
(1969)
Developmental screening 0-5 years, clinics in developmental medicine
-
-
Egan, D.F.1
Illingworth, R.S.2
Mac Keith, R.C.3
-
31
-
-
48249153186
-
Intraclass correlations: uses in assessing rater reliability
-
Shrout P.E., and Fleiss J.L. Intraclass correlations: uses in assessing rater reliability. Psychol Bull 86 (1979) 420-428
-
(1979)
Psychol Bull
, vol.86
, pp. 420-428
-
-
Shrout, P.E.1
Fleiss, J.L.2
-
33
-
-
0025500319
-
The process of rater training for observational instruments: implications for interrater reliability
-
Castorr A.H., Thompson K.O., Ryan J.W., Phillips C.Y., Prescott P.A., and Soeken K.L. The process of rater training for observational instruments: implications for interrater reliability. Res Nurs Health 13 5 (1990) 311-318
-
(1990)
Res Nurs Health
, vol.13
, Issue.5
, pp. 311-318
-
-
Castorr, A.H.1
Thompson, K.O.2
Ryan, J.W.3
Phillips, C.Y.4
Prescott, P.A.5
Soeken, K.L.6
-
34
-
-
0023229891
-
Interrater reliability: essential for research and practice
-
Haller K.B. Interrater reliability: essential for research and practice. MCN Am J Mater Child Nurs 12 1 (1987) 78
-
(1987)
MCN Am J Mater Child Nurs
, vol.12
, Issue.1
, pp. 78
-
-
Haller, K.B.1
-
35
-
-
0024115223
-
Interrater reliability: the underdeveloped role of rater training
-
Ryan J.W., Phillips C.Y., and Prescott P.A. Interrater reliability: the underdeveloped role of rater training. Appl Nurs Res 1 3 (1988) 148-150
-
(1988)
Appl Nurs Res
, vol.1
, Issue.3
, pp. 148-150
-
-
Ryan, J.W.1
Phillips, C.Y.2
Prescott, P.A.3
-
36
-
-
84988113075
-
Arthritis hand function test: inter-rater reliability among self-trained raters
-
Backman C., and Mackie H. Arthritis hand function test: inter-rater reliability among self-trained raters. Arthritis Care Res 8 1 (1995) 10-15
-
(1995)
Arthritis Care Res
, vol.8
, Issue.1
, pp. 10-15
-
-
Backman, C.1
Mackie, H.2
-
37
-
-
0023907516
-
Pragmatic aspects of establishing interrater reliability in research
-
Washington C.C., and Moss M. Pragmatic aspects of establishing interrater reliability in research. Nurs Res 37 3 (1988) 190-191
-
(1988)
Nurs Res
, vol.37
, Issue.3
, pp. 190-191
-
-
Washington, C.C.1
Moss, M.2
|