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Off-Label Use of Ataluren in Four Non-ambulatory Patients With Duchenne Muscular Dystrophy: Effects on Cardiac and Pulmonary Function and Muscle Strength
About 15% of Duchenne muscular dystrophy (DMD) cases are caused by point mutations leading to premature stop codons and disrupted synthesis of the dystrophin protein. Stop codon read-through therapy is available with the drug Ataluren (Translarna® by PTC Therapeutics). Following positive results in...
में बचाया:
| में प्रकाशित: | Front Pediatr |
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| मुख्य लेखकों: | , , , , , , , , |
| स्वरूप: | Artigo |
| भाषा: | Inglês |
| प्रकाशित: |
Frontiers Media S.A.
2018
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| विषय: | |
| ऑनलाइन पहुंच: | https://ncbi.nlm.nih.gov/pmc/articles/PMC6206203/ https://ncbi.nlm.nih.gov/pubmed/30406066 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.3389/fped.2018.00316 |
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