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- Casimersen, sold under the brand name Amondys 45, is an antisense oligonucleotide medication used for the treatment of Duchenne muscular dystrophy (DMD) in people who have a confirmed mutation of the dystrophin gene that is amenable to exon 45 skipping. It is an antisense oligonucleotide of phosphorodiamidate morpholino oligomer (PMO). Duchenne muscular dystrophy is a rare disease that primarily affects boys. It is caused by low levels of a muscle protein called dystrophin. The lack of dystrophin causes progressive muscle weakness and premature death. The most common side effects include upper respiratory tract infections, cough, fever, headache, joint pain and throat pain. Casimersen was approved for medical use in the United States in February 2021, and it is the first FDA-approved targeted treatment for people who have a confirmed mutation of the DMD gene that is amenable to skipping exon 45. (en)
- Casimersen ist ein Arzneistoff aus der Gruppe der Antisense-Therapeutika. Als Amondys 45 wurde es im Februar 2021 in den USA zur Behandlung einer bestimmten genetischen Ausprägung der Duchenne-Muskeldystrophie (DMD) zugelassen. (de)
- Касимерсен — лекарственный препарат для лечения мышечной дистрофии Дюшенна. Одобрен для применения: США (2021). (ru)
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- Casimersen ist ein Arzneistoff aus der Gruppe der Antisense-Therapeutika. Als Amondys 45 wurde es im Februar 2021 in den USA zur Behandlung einer bestimmten genetischen Ausprägung der Duchenne-Muskeldystrophie (DMD) zugelassen. (de)
- Касимерсен — лекарственный препарат для лечения мышечной дистрофии Дюшенна. Одобрен для применения: США (2021). (ru)
- Casimersen, sold under the brand name Amondys 45, is an antisense oligonucleotide medication used for the treatment of Duchenne muscular dystrophy (DMD) in people who have a confirmed mutation of the dystrophin gene that is amenable to exon 45 skipping. It is an antisense oligonucleotide of phosphorodiamidate morpholino oligomer (PMO). Duchenne muscular dystrophy is a rare disease that primarily affects boys. It is caused by low levels of a muscle protein called dystrophin. The lack of dystrophin causes progressive muscle weakness and premature death. (en)
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- Casimersen (en)
- Casimersen (de)
- Касимерсен (ru)
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