G.P.1.06 Outcome of Duchenne muscular dystrophy patients treated with daily deflazacort, daily prednisone, low dose 10days on/10days off prednisone and high dose weekend prednisone

2007 ◽  
Vol 17 (9-10) ◽  
pp. 773-774
Author(s):  
J. Collins ◽  
M. Knue ◽  
C. Wang ◽  
K. Kinnett ◽  
M. Kalra ◽  
...  
Keyword(s):  
Duchenne Muscular Dystrophy ◽  
Muscular Dystrophy ◽  
Low Dose ◽  
High Dose ◽  
Daily Prednisone

scholarly journals A phase 3 randomized placebo-controlled trial of tadalafil for Duchenne muscular dystrophy

Neurology ◽  
2017 ◽  
Vol 89 (17) ◽  
pp. 1811-1820 ◽  
Author(s):  
Ronald G. Victor ◽  
H. Lee Sweeney ◽  
Richard Finkel ◽  
Craig M. McDonald ◽  
Barry Byrne ◽  
...  
Keyword(s):  
Duchenne Muscular Dystrophy ◽  
Muscular Dystrophy ◽  
Upper Limb ◽  
Low Dose ◽  
Controlled Trial ◽  
High Dose ◽  
Ambulatory Ability ◽  
Efficacy Measure ◽  
Efficacy Measures ◽  
Primary Hypothesis

Objective:To conduct a randomized trial to test the primary hypothesis that once-daily tadalafil, administered orally for 48 weeks, lessens the decline in ambulatory ability in boys with Duchenne muscular dystrophy (DMD).Methods:Three hundred thirty-one participants with DMD 7 to 14 years of age taking glucocorticoids were randomized to tadalafil 0.3 mg·kg−1·d−1, tadalafil 0.6 mg·kg−1·d−1, or placebo. The primary efficacy measure was 6-minute walk distance (6MWD) after 48 weeks. Secondary efficacy measures included North Star Ambulatory Assessment and timed function tests. Performance of Upper Limb (PUL) was a prespecified exploratory outcome.Results:Tadalafil had no effect on the primary outcome: 48-week declines in 6MWD were 51.0 ± 9.3 m with placebo, 64.7 ± 9.8 m with low-dose tadalafil (p= 0.307 vs placebo), and 59.1 ± 9.4 m with high-dose tadalafil (p= 0.538 vs placebo). Tadalafil also had no effect on secondary outcomes. In boys >10 years of age, total PUL score and shoulder subscore declined less with low-dose tadalafil than placebo. Adverse events were consistent with the known safety profile of tadalafil and the DMD disease state.Conclusions:Tadalafil did not lessen the decline in ambulatory ability in boys with DMD. Further studies should be considered to confirm the hypothesis-generating upper limb data and to determine whether ambulatory decline can be slowed by initiation of tadalafil before 7 years of age.Clinicaltrials.gov identifier:NCT01865084.Classification of evidence:This study provides Class I evidence that tadalafil does not slow ambulatory decline in 7- to 14-year-old boys with Duchenne muscular dystrophy.

T.P.49 Low-dose steroid therapy for cardiomyopathy in Duchenne Muscular Dystrophy patients

2012 ◽  
Vol 22 (9-10) ◽  
pp. 866
Author(s):  
K. Ishigaki ◽  
T. Murakami ◽  
T. Saito ◽  
T. Sato ◽  
S. Kajino ◽  
...  
Keyword(s):  
Duchenne Muscular Dystrophy ◽  
Muscular Dystrophy ◽  
Steroid Therapy ◽  
Low Dose

scholarly journals Randomized, blinded trial of weekend vs daily prednisone in Duchenne muscular dystrophy

Neurology ◽  
2011 ◽  
Vol 77 (5) ◽  
pp. 444-452 ◽  
Author(s):  
D. M. Escolar ◽  
L. P. Hache ◽  
P. R. Clemens ◽  
A. Cnaan ◽  
C. M. McDonald ◽  
...  
Keyword(s):  
Duchenne Muscular Dystrophy ◽  
Muscular Dystrophy ◽  
Daily Prednisone ◽  
Blinded Trial

P4.6 Efficacy of low-dose steroid therapy for scoliosis in patients with Duchenne muscular dystrophy

2011 ◽  
Vol 21 (9-10) ◽  
pp. 706
Author(s):  
T. Murakami ◽  
K. Ishigaki ◽  
T. Saito ◽  
T. Sato ◽  
S. Onai ◽  
...  
Keyword(s):  
Duchenne Muscular Dystrophy ◽  
Muscular Dystrophy ◽  
Steroid Therapy ◽  
Low Dose

Daily prednisone treatment in duchenne muscular dystrophy in southwest china

2015 ◽  
Vol 52 (6) ◽  
pp. 1001-1007 ◽  
Author(s):  
Jun Hu ◽  
Yuanzhen Ye ◽  
Min Kong ◽  
Siqi Hong ◽  
Li Cheng ◽  
...  
Keyword(s):  
Duchenne Muscular Dystrophy ◽  
Muscular Dystrophy ◽  
Southwest China ◽  
Prednisone Treatment ◽  
Daily Prednisone

High dose weekly oral prednisone improves strength in boys with Duchenne muscular dystrophy

2002 ◽  
Vol 12 (10) ◽  
pp. 917-925 ◽  
Author(s):  
Anne M. Connolly ◽  
Jeanine Schierbecker ◽  
Renee Renna ◽  
Julaine Florence
Keyword(s):  
Duchenne Muscular Dystrophy ◽  
Muscular Dystrophy ◽  
Oral Prednisone ◽  
High Dose
Sign in / Sign up

Export Citation Format

Share Document