On their family farm in rural Illinois, the Flessner boys have free range. But early on, something was holding older brother ...

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

The critical health benefit for millions of Americans looked to be all set for a two year extension. Then Elon and Trump ...

Patients with Duchenne muscular dystrophy now have new hope with gene therapy. And for an Illinois family, it’s a double ...

From imaging innovations to new treatment approvals, 2024 brought advancements in Duchenne muscular dystrophy care and ...

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。

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 ...

Columnist Shalom Lim appreciates how Duchenne never offers a dull moment as he prepares for the challenges and adventures in ...

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

Patients with Duchenne muscular dystrophy now have new hope with gene therapy. And for an Illinois family, it’s a double blessing. On their family farm in rural Illinois, the Flessner boys have ...

Wedbush initiated coverage on Solid Biosciences, Inc. (NASDAQ:SLDB), a gene therapy company focused on treating Duchenne ...