VYONDYS 53 is an FDA-approved Duchenne muscular dystrophy treatment for patients who have a confirmed genetic mutation in the dystrophin gene that can be treated by skipping exon 53. In some patients, it helps the body make a shorter form of the dystrophin protein.

VYONDYS 53 is an antisense oligonucleotide indicated for the treatment of Duchenne muscular dystrophy (DMD) in patients who have a confirmed mutation of the DMD gene that is amenable to...

2019年12月16日 · 近日，该公司宣布，美国食品和药物管理局（FDA）已加速批准Vyondys 53（golodirsen），用于治疗经检测证实适合使用外显子53跳跃（exon 53 skipping）治疗的杜氏肌营养不良症（DMD）患. 2019年12月16日讯 / 生物谷 BIOON/ --Sarepta Therapeutics是一家专注于开发精准基因疗法治疗罕见病的生物技术公司。 近日，该公司宣布，美国食品和药物管理局（FDA）已加速批准Vyondys 53（golodirsen），用于治疗经检测证实适合使用外显子53跳 …

The U.S. Food and Drug Administration today granted accelerated approval to Vyondys 53 (golodirsen) injection to treat Duchenne muscular dystrophy (DMD) patients who have a confirmed mutation...

今日，美国FDA宣布，加速批准Sarepta Therapeutics公司的 Vyondys 53（golodirsen）上市，治疗适于使用外显子53跳跃（exon 53 skipping）治疗的杜兴氏肌营养不良症（DMD）患者。 这些患者大约占DMD患者总数的8%。 Sa…

Vyondys 53 (golodirsen)是一种外显子跳跃治疗药物，促进杜氏肌营养不良症 (DMD)患者缺失的外显子 (特别是外显子53)的剪切和连接，能够在翻译过程中产生相对较小的但功能更完整的蛋白质，帮助修复DMD基因突变所产生的异常蛋白质。

VYONDYS 53 is indicated for the treatment of Duchenne muscular dystrophy (DMD) in patients who have a confirmed mutation of the DMD gene that is amenable to exon 53 skipping. This indication is approved under accelerated approval based on an increase in dystrophin production in skeletal muscle observed in patients treated with VYONDYS 53.

VYONDYS 53 is used to treat patients with Duchenne muscular dystrophy (DMD) who have a confirmed mutation in the dystrophin gene that can be treated by skipping exon 53. This indication is approved under accelerated approval based on an increase in dystrophin production in skeletal muscle observed in patients treated with VYONDYS 53.

2025年3月11日 · Vyondys 53 is used to treat Duchenne muscular dystrophy (DMD) in patients with a confirmed mutation of the DMD gene that is amenable to exon 53 skipping. It is given …

2025年3月13日 · VYONDYS 53 is indicated for the treatment of Duchenne muscular dystrophy (DMD) in patients who have a confirmed mutation of the DMD gene that is amenable to exon 53 skipping.