A Pfizer result is unsettling the gene therapy push for Duchenne muscular dystrophy: The boys didn't improve even though a ...

“神经肌肉疾病的基因治疗时代已经开始，这些疾病的复杂性要求我们不断创新，以改进针对肌肉的候选药物。我们设计的新一代基因治疗载体在疗效和安全性方面改变了游戏规则。目前正在对各种神经肌肉疾病进行测试，”Genethon研究所进行性肌肉萎缩症研究主任兼负责人Isabelle Richard说。

Confounding data from a Pfizer clinical trial have rattled the field of gene therapy for Duchenne muscular dystrophy ...

Despite uncertainty around who will sit in The White House in January and what ramifications they may bring, the industry’s ...

SGT-003 utilises a novel muscle-tropic AAV capsid (AAV-SLB101) for delivery of Solid’s proprietary and differentiated neuronal nitric oxide synthase (nNOS) microdystrophin protein. In the meanti ...

Biopsies taken from the first three patients dosed with the treatment showed they had robust levels of microdystrophin, producing 38% of the protein compared to normal. All patients also showed ...