Validation of a prognostic score for changes in six-minute walk distance (6MWD) in patients with Duchenne muscular dystrophy (DMD)

2017 ◽  
Vol 27 ◽  
pp. S232
Author(s):  
N. Goemans ◽  
M. vanden Hauwe ◽  
G. Sajeev ◽  
Z. Yao ◽  
E. McDonnell ◽  
...  
Keyword(s):  
Duchenne Muscular Dystrophy ◽  
Muscular Dystrophy ◽  
Prognostic Score ◽  
Walk Distance ◽  
Minute Walk Distance ◽  
Minute Walk

scholarly journals Categorizing natural history trajectories of ambulatory function measured by the 6-minute walk distance in patients with Duchenne muscular dystrophy

2016 ◽  
Vol 26 (9) ◽  
pp. 576-583 ◽  
Author(s):  
Eugenio Mercuri ◽  
James Edward Signorovitch ◽  
Elyse Swallow ◽  
Jinlin Song ◽  
Susan J. Ward ◽  
...  
Keyword(s):  
Duchenne Muscular Dystrophy ◽  
Muscular Dystrophy ◽  
Natural History ◽  
Walk Distance ◽  
Minute Walk Distance ◽  
Minute Walk

scholarly journals Individualized Prediction of Changes in 6-Minute Walk Distance for Patients with Duchenne Muscular Dystrophy

PLoS ONE ◽  
2016 ◽  
Vol 11 (10) ◽  
pp. e0164684 ◽  
Author(s):  
Nathalie Goemans ◽  
Marleen vanden Hauwe ◽  
James Signorovitch ◽  
Elyse Swallow ◽  
Jinlin Song ◽  
...  
Keyword(s):  
Duchenne Muscular Dystrophy ◽  
Muscular Dystrophy ◽  
Walk Distance ◽  
Individualized Prediction ◽  
Minute Walk Distance ◽  
Minute Walk

Ambulatory capacity and disease progression as measured by the 6-minute-walk-distance in Duchenne muscular dystrophy subjects on daily corticosteroids

2013 ◽  
Vol 23 (8) ◽  
pp. 618-623 ◽  
Author(s):  
Nathalie Goemans ◽  
Marleen van den Hauwe ◽  
Rosamund Wilson ◽  
Annelies van Impe ◽  
Katrijn Klingels ◽  
...  
Keyword(s):  
Duchenne Muscular Dystrophy ◽  
Muscular Dystrophy ◽  
Disease Progression ◽  
Walk Distance ◽  
Minute Walk Distance ◽  
Minute Walk

scholarly journals Percent-Predicted 6-Minute Walk Distance in Duchenne Muscular Dystrophy to Account for Maturational Influences

PLoS Currents ◽  
2012 ◽  
Vol 4 ◽  
pp. RRN1297 ◽  
Author(s):  
Erik Henricson ◽  
Richard Abresch ◽  
Jay J. Han ◽  
Alina Nicorici ◽  
Erica Goude Keller ◽  
...  
Keyword(s):  
Duchenne Muscular Dystrophy ◽  
Muscular Dystrophy ◽  
Walk Distance ◽  
Minute Walk Distance ◽  
Minute Walk

scholarly journals D.08 ACT DMD (Ataluren Confirmatory Trial in Duchenne Muscular Dystrophy): effect of Ataluren on timed function tests (TFT) in nonsense mutation (nm) DMD

2016 ◽  
Vol 43 (S2) ◽  
pp. S15-S15
Author(s):  
N Goemans ◽  
C Campbell ◽  
CM McDonald ◽  
T Voit ◽  
X Luo ◽  
...  
Keyword(s):  
Duchenne Muscular Dystrophy ◽  
Meta Analysis ◽  
Double Blind Study ◽  
Walk Distance ◽  
Phase 3 ◽  
Double Blind ◽  
Decline Phase ◽  
Minute Walk Distance ◽  
Minute Walk ◽  
Confirmatory Trial

Background: Ataluren is the first drug to treat the underlying cause of nmDMD. Methods: ACT DMD is a Phase 3, randomized, double-blind study. Males 7-16 years with nmDMD and a screening six-minute walk distance (6MWD) ≥150 m and <80%-predicted were randomized to ataluren 40 mg/kg/day or placebo for 48 weeks. A pre-specified subgroup included patients with baseline 6MWD 300-400 m. A meta-analysis of the overall ACT DMD population and the ‘ambulatory decline phase’ subgroup of the Phase 2b study (those patients meeting ACT DMD entry criteria) was pre-specified in the statistical plan. Results: In the overall ACT DMD population (N=228), changes in TFTs favored ataluren over placebo: 10-meter walk/run, -1.2s (p=0.117); 4-stair climb, -1.8s (p=0.058); 4-stair descend, -1.8s (p=0.012). In the pre-specified subgroup (n=99), these differences increased to -2.1s, -3.6s, and -4.3s, respectively, and were statistically significant (p<0.01) for 4-stair climb and descend. Results are supported by the meta-analysis (N=291), which demonstrated significant differences (p<0.05) in 10-meter walk/run, 4-stair climb, 4-stair descend. Conclusions: TFT results showed a benefit for ataluren in ACT DMD, and a larger treatment effect in the pre-specified baseline 6MWD 300-400 m subgroup as well as the pre-specified meta-analysis of ACT DMD and the Phase 2b study decline subgroup.Supported By: PTC Therapeutics Inc.

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