-
2
-
-
70350046640
-
Prevalence of Duchenne/Becker muscular dystrophy among males aged 5-24 years: Four states 2007
-
Center for Disease Control and Prevention (CDC)
-
Center for Disease Control and Prevention (CDC). Prevalence of Duchenne/Becker muscular dystrophy among males aged 5-24 years: four states, 2007. MMWR Morb Mortal Wkly Rep 2009;58:1119-1122.
-
(2009)
MMWR Morb Mortal Wkly Rep
, vol.58
, pp. 1119-1122
-
-
-
3
-
-
0026344234
-
Long-term benefit from prednisone therapy in Duchenne muscular dystrophy
-
Fenichel GM, Florence JM, Pestronk A, et al. Long-term benefit from prednisone therapy in Duchenne muscular dystrophy. Neurology 1991;41:1874-1877.
-
(1991)
Neurology
, vol.41
, pp. 1874-1877
-
-
Fenichel, G.M.1
Florence, J.M.2
Pestronk, A.3
-
4
-
-
72149108443
-
Diagnosis and management of duchenne muscular dystrophy part 1: Diagnosis and pharmacological and psychosocial management
-
Bushby K, Finkel R, Birnkrant DJ, et al. Diagnosis and management of Duchenne muscular dystrophy, part 1: diagnosis, and pharmacological and psychosocial management. Lancet Neurol 2010;9;77-93.
-
(2010)
Lancet Neurol
, vol.9
, pp. 77-93
-
-
Bushby, K.1
Finkel, R.2
Birnkrant, D.J.3
-
5
-
-
76549130473
-
Diagnosis and management of duchenne muscular dystrophy part 2: Implementation of multidisciplinary care
-
Bushby K, Finkel R, Birnkrant DJ, et al. Diagnosis and management of Duchenne muscular dystrophy, part 2: implementation of multidisciplinary care. Lancet Neurol 2010;9:177-189.
-
(2010)
Lancet Neurol
, vol.9
, pp. 177-189
-
-
Bushby, K.1
Finkel, R.2
Birnkrant, D.J.3
-
7
-
-
78650186613
-
Use of cortico-steroids in a population based cohort of boys with Duchenne and Becker muscular dystrophy
-
Matthews DJ, James KA, Miller LA, et al. Use of cortico-steroids in a population based cohort of boys with Duchenne and Becker muscular dystrophy. J Child Neu-rol 2010;25:1319-1324.
-
(2010)
J Child Neu-rol
, vol.25
, pp. 1319-1324
-
-
Matthews, D.J.1
James, K.A.2
Miller, L.A.3
-
9
-
-
84856208646
-
-
Hilbert JE, Kissel JT, Luebbe EA, et al. If you build a rare disease registry, will they enroll and will they use it? Methods and data from the National Registry of Myotonic Dystrophy (DM) and Facioscapulohumeral Muscular Dystrophy (FSHD). Contemp Clin Trials 2012;33:302-311.
-
(2012)
If You Build A Rare Disease Registry, Will They Enroll and Will They Use It? Methods and Data from the National Registry of Myotonic Dystrophy (DM) and Facioscapulohumeral Muscular Dystrophy (FSHD). Contemp Clin Trials
, vol.33
, pp. 302-311
-
-
Hilbert, J.E.1
Kissel, J.T.2
Luebbe, E.A.3
-
10
-
-
77952420292
-
Improving the quality of care for patients with cystic fibrosis
-
Schechter MS, Gutierrez HH. Improving the quality of care for patients with cystic fibrosis. Curr Opin Pediatr 2010;22:296-301.
-
(2010)
Curr Opin Pediatr
, vol.22
, pp. 296-301
-
-
Schechter, M.S.1
Gutierrez, H.H.2
-
11
-
-
25144516833
-
Improving subspecialty healthcare: Lessons from cystic fibrosis
-
Schechter MS, Margolis P. Improving subspecialty healthcare: lessons from cystic fibrosis. J Pediatr 2005;147:295-301.
-
(2005)
J Pediatr
, vol.147
, pp. 295-301
-
-
Schechter, M.S.1
Margolis, P.2
-
12
-
-
81555201440
-
A story of success: Continuous quality improvement in cystic fibrosis care in the usa
-
Quon BS, Goss C. A story of success: continuous quality improvement in cystic fibrosis care in the USA. Thorax 2011;66:1106-1108.
-
(2011)
Thorax
, vol.66
, pp. 1106-1108
-
-
Quon, B.S.1
Goss, C.2
-
13
-
-
56249132492
-
Patient registry analyses: Seize the data, but caveat lector
-
Schechter MS. Patient registry analyses: seize the data, but caveat lector. J Pediatr 2008;153:733-755.
-
(2008)
J Pediatr
, vol.153
, pp. 733-755
-
-
Schechter, M.S.1
-
14
-
-
84855168553
-
-
Cystic Fibrosis Foundation Patent Registry Accessed February 7, 2012
-
Cystic Fibrosis Foundation Patent Registry. Annual data report 2010. Available at: http://www.cff.org/UploadedFiles/LivingWithCF/CareCenterNetwork/ PatientRegistry/2010-Patient-Registry-Report.pdf. Accessed February 7, 2012.
-
Annual Data Report 2010
-
-
-
16
-
-
19944427852
-
Practice parameter: Corticosteroid treatment of Duchenne dystrophy: Report of the Quality Standards Subcommittee of the American Academy of Neurology and the Practice Committee of the Child Neurology Society
-
Moxley RT III, Ashwal S, Pandya S, et al. Practice parameter: corticosteroid treatment of Duchenne dystrophy: report of the Quality Standards Subcommittee of the American Academy of Neurology and the Practice Committee of the Child Neurology Society. Neurology 2005;64:13-20.
-
(2005)
Neurology
, vol.64
, pp. 13-20
-
-
Moxley III, R.T.1
Ashwal, S.2
Pandya, S.3
-
17
-
-
80155123446
-
Identification of research gaps from evidence-based guidelines: A pilot study in cystic fibrosis
-
Robinson KA, Saldanha IJ, Mckoy NA. Identification of research gaps from evidence-based guidelines: a pilot study in cystic fibrosis. Int J Technol Asses 2011;27:247-252.
-
(2011)
Int J Technol Asses
, vol.27
, pp. 247-252
-
-
Robinson, K.A.1
Saldanha, I.J.2
McKoy, N.A.3
-
18
-
-
84873696600
-
-
Accessed February 7, 2012
-
Cystic Fibrosis Care Guidelines. Available at: http://www.cff.org/ treatments/CFCareGuidelines/. Accessed February 7, 2012.
-
Cystic Fibrosis Care Guidelines
-
-
-
19
-
-
27144479427
-
Development and validation of the Cystic Fibrosis Questionnaire in the United States: A health related qual-ity-of-life measure for cystic fibrosis
-
Quittner AL, Buu A, Messer MA, Modi AC, Watrous M. Development and validation of The Cystic Fibrosis Questionnaire in the United States: a health related qual-ity-of-life measure for cystic fibrosis. Chest 2005;128:2347-2354.
-
(2005)
Chest
, vol.128
, pp. 2347-2354
-
-
Quittner, A.L.1
Buu, A.2
Messer, M.A.3
Modi, A.C.4
Watrous, M.5
-
21
-
-
80052840993
-
-
Cystic Fibrosis Foundation Accessed February 7, 2012
-
Cystic Fibrosis Foundation. 2010 annual report. Available at: http://www.cff.org/UploadedFiles/aboutCFFoundation/AnnualReport/ 2010-Annual-Report.pdf. Accessed February 7, 2012.
-
2010 Annual Report
-
-
-
22
-
-
34249099687
-
Managing Duchenne muscular dystrophy: The additive effect of spinal surgery and home nocturnal ventilation in improving survival
-
Eagle M, Bourke J, Bullock R, et al. Managing Duchenne muscular dystrophy: the additive effect of spinal surgery and home nocturnal ventilation in improving survival. Neuromuscul Disord 2007;17:470-475.
-
(2007)
Neuromuscul Disord
, vol.17
, pp. 470-475
-
-
Eagle, M.1
Bourke, J.2
Bullock, R.3
-
23
-
-
0036895043
-
Survival in Duchenne muscular dystrophy: Improvements in life expectancy since 1967 and the impact of home nocturnal ventilation
-
Eagle M, Baudouin SV, Chandler C, et al. Survival in Duchenne muscular dystrophy: improvements in life expectancy since 1967 and the impact of home nocturnal ventilation. Neuromuscul Disord 2002;12:926-929.
-
(2002)
Neuromuscul Disord
, vol.12
, pp. 926-929
-
-
Eagle, M.1
Baudouin, S.V.2
Chandler, C.3
-
24
-
-
77956325548
-
Muscular Dystrophy Surveillance Tracking and Research Network (MD STARnet): Case definitions in surveillance for childhood-onset Duchenne/Becker muscular dystrophy
-
Mathews KD, Cunniff C, Kantamneni JR, et al. Muscular Dystrophy Surveillance Tracking and Research Network (MD STARnet): case definitions in surveillance for childhood-onset Duchenne/Becker muscular dystrophy. J Child Neurol 2010;25:1089-1102.
-
(2010)
J Child Neurol
, vol.25
, pp. 1089-1102
-
-
Mathews, K.D.1
Cunniff, C.2
Kantamneni, J.R.3
-
25
-
-
33751174484
-
The Muscular Dystrophy Surveillance Tracking and Research Network (MD STARnet): Surveillance methodology: Birth defects research (part A)
-
Miller LA, Romitti PA, Cunniff C, et al. The Muscular Dystrophy Surveillance Tracking and Research Network (MD STARnet): surveillance methodology: birth defects research (part A). Clin Mol Technol 2006;76:793-797.
-
(2006)
Clin Mol Technol
, vol.76
, pp. 793-797
-
-
Miller, L.A.1
Romitti, P.A.2
Cunniff, C.3
-
26
-
-
70349599869
-
Collaborating to bring new therapies to the patient: The TREAT-NMD model
-
Bushby K, Lynn S, Straub V. Collaborating to bring new therapies to the patient: the TREAT-NMD model. Acta Myo 2009;28:12-15.
-
(2009)
Acta Myo
, vol.28
, pp. 12-15
-
-
Bushby, K.1
Lynn, S.2
Straub, V.3
-
27
-
-
84873619703
-
-
Charter for TREAT-NMD patient database/registry Accessed February 7, 2012
-
Charter for TREAT-NMD patient database/registry. Available at: http://www.treat-nmd.eu/downloads/file/registries-toolkit/charter-treat-nmd- global-database- approved.pdf. Accessed February 7, 2012.
-
-
-
-
29
-
-
84873654275
-
-
CARE-NMD Accessed February 7, 2012
-
CARE-NMD. Available at: http://en.care-nmd.eu/. Accessed February 7, 2012.
-
-
-
-
30
-
-
84873649351
-
-
DuchenneConnect Accessed February 7, 2012
-
DuchenneConnect. Available at: https://www.duchenneconnect. org/. Accessed February 7, 2012.
-
-
-
-
31
-
-
84873679726
-
DuchenneConnect registry report [Internet]. Version 17
-
Accessed March 30, 2012
-
Rangel V, Martin AS, Peay HL. DuchenneConnect registry report [Internet]. Version 17. PLoS currents: Muscular Dystrophy. Available at: http://knol.google.com/k/vanessa-rangel/duchenneconnect-registry-report/ 3c8kwqirmpdbh/3. Accessed March 30, 2012.
-
PLoS Currents: Muscular Dystrophy
-
-
Rangel, V.1
Martin, A.S.2
Peay, H.L.3
-
32
-
-
84873626602
-
-
Muscular Dystrophy Association Accessed March 30, 2012
-
Muscular Dystrophy Association. Available at: http://www.mdausa.org/. Accessed March 30, 2012.
-
-
-
-
33
-
-
84873627165
-
-
Muscular Dystrophy Association Clinical Conference Information Accessed on March 30, 2012
-
Muscular Dystrophy Association Clinical Conference Information. Available at: http://www.mdausa.org/research/national-conferences/2012-clinical- conference/information. htm. Accessed on March 30, 2012.
-
-
-
-
34
-
-
77957267994
-
Widening gap in age at muscular dystrophy-associated death between blacks and whites 1986-2005
-
Kenneson A, Vatave A, Finkel R. Widening gap in age at muscular dystrophy-associated death between blacks and whites, 1986-2005. Neurology 2010;75: 982-989.
-
(2010)
Neurology
, vol.75
, pp. 982-989
-
-
Kenneson, A.1
Vatave, A.2
Finkel, R.3
-
35
-
-
81155161042
-
Disparities in the diagnostic process of duchenne and becker muscular dystrophy
-
Holtzer C, Meaney FJ, Andrews J, et al. Disparities in the diagnostic process of Duchenne and Becker muscular dystrophy. Genet Med 2011;13:942-947.
-
(2011)
Genet Med
, vol.13
, pp. 942-947
-
-
Holtzer, C.1
Meaney, F.J.2
Andrews, J.3
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