التفاصيل البيبلوغرافية
| العنوان: |
Ataluren Preserves Muscle Function in nmDMD Patients: A Pooled Analysis of Results from Three Randomized, Double-Blind, Placebo-Controlled Trials. |
| المؤلفون: |
McDonald, C. M., Jong, Y.-J., Karachunski, P., Statland, J., Lorentzos, M., Cairns, A., Takeshima, Y., Penematsa, V., Chou, C., Lin, M., Werner, C., Trifillis, P., Gordon, G., Williams, P., Grünert, J., Klein, M., Bramlage, P. |
| المصدر: |
Neuropediatrics; 2023 Supplement 1, Vol. 54, pS1-S32, 32p |
| مصطلحات موضوعية: |
DUCHENNE muscular dystrophy, NONSENSE mutation |
| مستخلص: |
This article, published in the journal Neuropediatrics, presents the results of a pooled analysis of three randomized, double-blind, placebo-controlled trials of Ataluren in patients with nonsense mutation Duchenne muscular dystrophy (nmDMD). The study included a total of 701 patients and assessed the efficacy of Ataluren in preserving muscle function. The results showed that Ataluren significantly reduced the decline in muscle function compared to placebo, as measured by various tests including the 6-minute walk distance and timed function tests. These findings suggest that Ataluren may be effective in preserving muscle function in patients with nmDMD. [Extracted from the article] |
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| قاعدة البيانات: |
Complementary Index |
