The embryos were female, and Jenssen remembers the doctors assuring her that, because the Duchenne mutation is linked to the ...

About Solid Biosciences Solid Biosciences is a precision genetic medicine company focused on advancing a portfolio of gene ...

A possible gene therapy approval and a series of major trials readouts set the stage for a busy year of drug development in ...

The Billings Livestock Commission hosted the sixth annual Calves to Cure to help raise money and awareness for Duchenne ...

Michelle C. Werner agonized over whether the gene therapy treatment Elevidys was right for her teenage son. But to him, the ...

An important subgroup are the dystrophin-related muscular dystrophies caused by variants in the DMD gene. They can be divided into the more severe Duchenne muscular dystrophy and the milder Becker ...

Although I’ve been a member of SPAN since 2022, this event was the first time I’d become actively involved in its advocacy ...

Neu refix granted orphan disease designation (ODD) & rare pediatric disease designation (RPDD) by US FDA for treatment of ...

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。

The FDA has granted Orphan Drug and Rare Pediatric Disease Designations to Somite’s cell replacement therapy for the ...

Six-year-old Cooper Wood was diagnosed with Duchenne muscular dystrophy earlier this year. When his parents applied for a one ...