A phase 2 trial of the safety and pharmacokinetics of ataluren in patients aged ≥2 to <5 years with nonsense mutation Duchenne muscular dystrophy

2018 ◽  
Vol 28 ◽  
pp. S12-S13
Author(s):  
R. Kong ◽  
E. O’Mara ◽  
X. Luo ◽  
P. Trifillis ◽  
C. Werner ◽  
...  
Keyword(s):  
Duchenne Muscular Dystrophy ◽  
Muscular Dystrophy ◽  
Nonsense Mutation ◽  
Phase 2 ◽  
Phase 2 Trial

scholarly journals Early phase 2 trial of TAS‐205 in patients with Duchenne muscular dystrophy

2020 ◽  
Vol 7 (2) ◽  
pp. 181-190 ◽  
Author(s):  
Hirofumi Komaki ◽  
Yoshihiro Maegaki ◽  
Tsuyoshi Matsumura ◽  
Kazuhiro Shiraishi ◽  
Hiroyuki Awano ◽  
...  
Keyword(s):  
Duchenne Muscular Dystrophy ◽  
Muscular Dystrophy ◽  
Early Phase ◽  
Phase 2 ◽  
Phase 2 Trial

scholarly journals Placebo-controlled Phase 2 Trial of Drisapersen for Duchenne Muscular Dystrophy

2018 ◽  
Vol 5 (8) ◽  
pp. 913-926 ◽  
Author(s):  
Craig M. McDonald ◽  
Brenda Wong ◽  
Kevin M. Flanigan ◽  
Rosamund Wilson ◽  
Sjef de Kimpe ◽  
...  
Keyword(s):  
Duchenne Muscular Dystrophy ◽  
Muscular Dystrophy ◽  
Phase 2 ◽  
Phase 2 Trial

scholarly journals Randomized phase 2 trial and open-label extension of domagrozumab in Duchenne muscular dystrophy

2020 ◽  
Vol 30 (6) ◽  
pp. 492-502 ◽  
Author(s):  
Kathryn R. Wagner ◽  
Hoda Z. Abdel-Hamid ◽  
Jean K. Mah ◽  
Craig Campbell ◽  
Michela Guglieri ◽  
...  
Keyword(s):  
Duchenne Muscular Dystrophy ◽  
Muscular Dystrophy ◽  
Phase 2 ◽  
Open Label ◽  
Phase 2 Trial ◽  
Open Label Extension ◽  
Randomized Phase 2

G.P.3.05 Phase 2 study of PTC124 for nonsense mutation suppression therapy of Duchenne muscular dystrophy (DMD)

2007 ◽  
Vol 17 (9-10) ◽  
pp. 783 ◽  
Author(s):  
C. Bönnemann ◽  
R. Finkel ◽  
B. Wong ◽  
K. Flanigan ◽  
J. Sampson ◽  
...  
Keyword(s):  
Duchenne Muscular Dystrophy ◽  
Muscular Dystrophy ◽  
Nonsense Mutation ◽  
Phase 2 ◽  
Phase 2 Study

MoveDMD: phase 2 trial of edasalonexent, an NF-κB inhibitor, in 4 to 7-year old patients with Duchenne muscular dystrophy

2017 ◽  
Vol 27 ◽  
pp. S215
Author(s):  
R. Finkel ◽  
K. Vandenborne ◽  
H. Sweeney ◽  
E. Finanger ◽  
G. Tennekoon ◽  
...  
Keyword(s):  
Duchenne Muscular Dystrophy ◽  
Muscular Dystrophy ◽  
Phase 2 ◽  
Old Patients ◽  
Phase 2 Trial

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
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