"My medicine is making me stronger," 7-year-old Hudson Sanford says after receiving a breakthrough gene therapy for Duchenne ...

Discover six innovative clinical-stage biotech companies fostering R&D in the Duchenne muscular dystrophy therapeutic space.

The play is known as the Jackson Special. Kelly Maynard, Jackson’s mom, said he was diagnosed with Duchenne’s Muscular ...

Deb Jenssen never wanted her children to suffer from the disease that killed her brother at 28. The illness, Duchenne muscular dystrophy, initially manifests in childhood as trouble with strength and ...

A dispute over a Duchenne gene therapy highlights thorny issues surrounding FDA approvals and insurance policies.

日前，昆明理工大学灵长类转化医学研究院、省部共建非人灵长类国家重点实验室、昆明理工大学生命科学与技术学院陈永昌、季维智团队，与广州实验室、上海交通大学医学院附属新华医院等机构合作，应用非人灵长类疾病模型解析杜氏肌营养不良症（Duchenne muscular dystrophy，DMD）发病早期骨骼肌病理变化的关键机制，在国际 ...

动物模型是疾病机制研究和药物开发的重要工具，然而目前已有的小鼠、狗、猪等DMD动物模型均不能很好模拟DMD患者的病程进展。昆明理工大学陈永昌/季维智团队经过多年努力，成功培育出F1代DMD猕猴模型，其中雄性半合子突变个体模拟DMD患者基因型，展现出与 ...

研究结果为DMD发病机制，特别是疾病早期的分子和细胞变化提供了新的见解，揭示了免疫、纤维化以及肌肉干细胞在DMD早期的动态变化，为早期干预和靶向治疗提供了科学依据。此外，DMD猴模型显示FAPs纤维化不依赖于TGFβ通路，为新药研发提供了新的方向。更重要的是，肌肉干细胞功能缺陷导致了肌肉修复障碍，提示DMD是一种干细胞疾病，开发细胞治疗或针对肌肉干细胞进行干预治疗也许是未来研究的重要方向。

Givinostat, is an HDAC inhibitor (HDACi, a principle candidate, currently being developed for the treatment of DMD and BMD.

Biotech reported positive interim data from a mid-stage trial of a treatment for the rare muscle disorder Duchenne muscular ...

A ‘Relive the 80s’ theme marked the occasion, which brought together 740 members of the commercial property industry at the ...

Capricor Therapeutics (NASDAQ: CAPR), a biotechnology company developing transformative cell and exosome-based therapeutics for the treatment of rare diseases, announced today that the Company will ...