Assessing the ability of boys with Duchenne muscular dystrophy age 4–7 years to swallow softgel capsules: Clinical trial experience with edasalonexent

2021 ◽  
Author(s):  
Maria Mancini ◽  
Gigi Shafai ◽  
Elizabeth Thaler ◽  
Joanne M. Donovan ◽  
Richard S. Finkel
Keyword(s):  
Clinical Trial ◽  
Duchenne Muscular Dystrophy ◽  
Muscular Dystrophy ◽  
Trial Experience ◽  
Clinical Trial Experience

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

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.

scholarly journals Comparable indicators of therapeutic misconception between epilepsy or Parkinson’s disease patients between those with clinical trial experience and trial non-participants

Seizure ◽  
2018 ◽  
Vol 60 ◽  
pp. 61-67 ◽  
Author(s):  
Emmi Reijula ◽  
Arja Halkoaho ◽  
Anna-Maija Pietilä ◽  
Tuomas Selander ◽  
Kirsti Martikainen ◽  
...  
Keyword(s):  
Parkinson’S Disease ◽  
Clinical Trial ◽  
Parkinson's Disease ◽  
Therapeutic Misconception ◽  
Trial Experience ◽  
Clinical Trial Experience

scholarly journals Correction to: Liver Safety of Fasiglifam (TAK-875) in Patients with Type 2 Diabetes: Review of the Global Clinical Trial Experience

Drug Safety ◽  
2018 ◽  
Vol 41 (12) ◽  
pp. 1431-1437 ◽  
Author(s):  
John F. Marcinak ◽  
Melvin S. Munsaka ◽  
Paul B. Watkins ◽  
Takashi Ohira ◽  
Neila Smith
Keyword(s):  
Type 2 Diabetes ◽  
Clinical Trial ◽  
Liver Safety ◽  
Trial Experience ◽  
Global Clinical Trial ◽  
Clinical Trial Experience

scholarly journals Expectations and anxieties of Duchenne muscular dystrophy patients and their families during the first-in-human clinical trial of NS-065/NCNP-01

2020 ◽  
Vol 42 (4) ◽  
pp. 348-356
Author(s):  
Reiko Shimizu ◽  
Maki Ohata ◽  
Hisateru Tachimori ◽  
En Kimura ◽  
Yuko Harada ◽  
...  
Keyword(s):  
Clinical Trial ◽  
Duchenne Muscular Dystrophy ◽  
Muscular Dystrophy ◽  
Human Clinical Trial
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