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The EC has granted orphan drug designation to Dyne Therapeutics’ DYNE-251 for treating Duchenne muscular dystrophy (DMD).

Dyne anticipates filing a Biologics License Application (BLA) submission for US accelerated approval in early 2026.

President Donald Trump told NBC News in an interview Saturday that he would not fire anyone involved in the Signal group chat in which military attack plans were inadvertently divulged to a ...

Explore GISTs, rare GI tumors. Discover key details on genetic causes, diagnostic methods, and cutting-edge treatment ...

It's been nearly two years since Elevidys, a gene therapy for Duchenne MD developed by Sarepta Therapeutics, was approved by ...

John Brandsema, MD, a pediatric neurologist at Children’s Hospital of Philadelphia, speaks with Healio about the current understanding of the genetic factors behind muscular dystrophy.

除了运动功能受损，肌营养不良还会引发一系列并发症。心脏受累是常见风险，部分患者会出现心肌病、心律失常；骨骼畸形也屡见不鲜，脊柱侧弯、关节挛缩严重影响患者的体态和活动能力。更残酷的是，许多患者因长期依赖轮椅，面临压疮、肺部感染等问题，生存质量每况愈下。

A trial found magnetic resonance imaging correlated with physical function tests in patients with Duchenne muscular dystrophy ...

Duchenne Muscular Dystrophy: diagnosis, clinical development and global researchMuscular dystrophy refers to a group of rare, genetic, progressive diseases that primarily affect the body’s muscles, ...

Two dose levels of a single-administration gene therapy were well-tolerated and led to functional improvements in ambulatory ...