Duchenne muscular dystrophy (DMD) is one of the most common and devastating childhood genetic disorders, affecting ...

EDO51, an investigational therapy for Duchenne muscular dystrophy (DMD), and is still ongoing in the United Kingdom.

Percheron Therapuetic's key trial for Duchenne muscular dystrophy has flopped, sending the shares into freefall ...

Sevasemten significantly reduced two markers of muscle damage in treated Becker muscular dystrophy patients in Phase 2 CANYON ...

The year is ending with a strong flow of clinical results, with Percheron Therapeutics due to release key trial data tomorrow ...

A novel drug holds promise for treating Duchenne muscular dystrophy (DMD), a rare genetic disorder that causes severe muscle ...

PepGen Inc. (Nasdaq: PEPG), a clinical-stage biotechnology company advancing the next generation of oligonucleotide therapies ...

SRP-5051 is an antisense oligonucleotide commercialized by Sarepta Therapeutics, with a leading Phase II program in Duchenne Muscular Dystrophy.

最初出现轻微运动障碍，跑步、跳跃等动作变得吃力，后期逐渐出现无法行走、肢体无法正常抬起，需要借助轮椅行动，最终可能因呼吸衰竭或心力衰竭而死亡……这段描述描写的是，杜氏肌营养不良症患者的一生。杜氏肌营养不良症（Duchenne Muscular ...

从微观领域，开发基因治疗方案也有了新线索。目前针对DMD患者的治疗手段非常有限，一种通用策略是通过基因治疗来修补功能异常的肌营养不良蛋白。但是，这个蛋白的基因是人体最长的基因之一，完整递送如此长的基因去替换致病基因十分困难。

北京时间2024年12月11日，西湖大学、西湖实验室吴建平团队联合闫浈团队在Nature在线发表题为“Structure and assembly of the dystrophin glycoprotein ...

Avidity Biosciences' AOC platform shows promising results for genetic diseases, making it a strong buy with potential upside ...