CINRG pilot trial of oxatomide in steroid-naïve Duchenne muscular dystrophy.
The authors report a pilot open-label two-center therapeutic trial of oxatomide in 14 steroid-naive DMD boys aged 5-10 years. Comparison of linear evolutions between 3 months medication-free lead-in periods and 6 months treatment periods showed no significant differences in quantitative (QMT) and manual (MMT) measurements of muscle strength and timed functional tests. A modest mitigation of strength deterioration over time cannot be excluded.
Buyse
Gunnar M
GM
University Hospitals KU Leuven, Leuven, Herestraat 49, B-3000 Leuven, Belgium. gunnar.buyse@uz.kuleuven.ac.be
Goemans
Nathalie
N
Henricson
Erik
E
Jara
Alejandro
A
van den Hauwe
Marleen
M
Leshner
Robert
R
Florence
Julaine M
JM
Mayhew
Jill E
JE
Escolar
Diana M
DM
D002648:Child; D002675:Child, Preschool; D005260:Female; D005500:Follow-Up Studies; D006634:Histamine H1 Antagonists / Q000627:therapeutic use*; D006801:Humans; D016014:Linear Models; D008297:Male; D053580:Muscle Strength / Q000187:drug effects; D020388:Muscular Dystrophy, Duchenne / Q000188:drug therapy*; D010865:Pilot Projects; D010879:Piperazines / Q000627:therapeutic use*; D013997:Time Factors; D016896:Treatment Outcome
D006634:Histamine H1 Antagonists; D010879:Piperazines; C015408:oxatomide