T.P.1 02 A phase I/II clinical trial in Duchenne muscular dystrophy using IM and IV delivered antisense oligonucleotides: The MDEX consortium

2006 ◽  
Vol 16 (9-10) ◽  
pp. 685 ◽  
Author(s):  
V. Arechavala ◽  
K. Bushby ◽  
G. Dickson ◽  
I. Graham ◽  
M. Kinali ◽  
...  
Keyword(s):  
Clinical Trial ◽  
Duchenne Muscular Dystrophy ◽  
Muscular Dystrophy ◽  
Phase I ◽  
Antisense Oligonucleotides

Ataluren: Clinical Trial Results in Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD)

2016 ◽  
Vol 47 (S 01) ◽  
Author(s):  
U. Schara ◽  
C. McDonald ◽  
K. Bushby ◽  
M. Tulinius ◽  
R. Finkel ◽  
...  
Keyword(s):  
Clinical Trial ◽  
Duchenne Muscular Dystrophy ◽  
Muscular Dystrophy ◽  
Nonsense Mutation ◽  
Clinical Trial Results

In vivocomparison of 2′-O-methyl phosphorothioate and morpholino antisense oligonucleotides for Duchenne muscular dystrophy exon skipping

2009 ◽  
Vol 11 (3) ◽  
pp. 257-266 ◽  
Author(s):  
Hans A. Heemskerk ◽  
Christa L. de Winter ◽  
Sjef J. de Kimpe ◽  
Petra van Kuik-Romeijn ◽  
Niki Heuvelmans ◽  
...  
Keyword(s):  
Duchenne Muscular Dystrophy ◽  
Muscular Dystrophy ◽  
Antisense Oligonucleotides ◽  
Exon Skipping

T.P.3.08 Clinical trial of epigallocatechin-3-gallate in Duchenne muscular dystrophy

2009 ◽  
Vol 19 (8-9) ◽  
pp. 612
Author(s):  
A. von Moers ◽  
F. Paul ◽  
M. Schülke ◽  
S. Ohlraum ◽  
Y. Nakae ◽  
...  
Keyword(s):  
Clinical Trial ◽  
Duchenne Muscular Dystrophy ◽  
Muscular Dystrophy

scholarly journals A.01 The relationship between fatigue and health-related quality of life in a clinical trial population of Duchenne muscular dystrophy patients

2017 ◽  
Vol 44 (S2) ◽  
pp. S8-S8
Author(s):  
Y Wei ◽  
B El-Aloul ◽  
C Nguyen ◽  
E Zapata-Aldana ◽  
C Campbell
Keyword(s):  
Quality Of Life ◽  
Clinical Trial ◽  
Duchenne Muscular Dystrophy ◽  
Muscular Dystrophy ◽  
Parent Report ◽  
Health Related Quality ◽  
Patient Report ◽  
Related Quality ◽  
Health Related

Background: Fatigue was recently reported to be the largest contributor to poor health-related quality of life (HRQOL) in paediatric Duchenne muscular dystrophy (DMD). Additional studies are necessary to confirm the generalizability of this finding. Our objective was to explore the longitudinal relationship between fatigue and HRQOL in an additional cohort of DMD patients. Methods: We performed a secondary analysis of data from a clinical trial (NCT00592553), which enrolled patients with nonsense mutation DMD, aged 5–20 years, from 37 sites in 11 countries (N=174). Fatigue and HRQOL were assessed using the PedsQLTM Multidimensional Fatigue Scale and Generic Core Scales, respectively, by patient- and parent-report at baseline and over 48 weeks. Results: Patients reported greater fatigue than healthy controls from published data. There was no significant difference between patient- and parent-reported fatigue. Fatigue was significantly correlated with worse HRQOL at baseline, by patient-report (r=0.70, P<0.001) and parent-report (r=0.70, P<0.001); and at 48 weeks, by patient-report (r=0.79, P<0.001) and parent-report (r=0.74, P<0.001). Change in fatigue was significantly correlated with change in HRQOL over 48 weeks, by patient-report (r=0.64, P<0.001) and parent-report (r=0.67, P<0.001). Conclusions: Fatigue is a major contributor to HRQOL in DMD. The strong association between fatigue and HRQOL corroborates previous studies, and suggests that reducing fatigue may improve HRQOL.

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