编辑丨王多鱼排版丨水成文衰老是所有成年人不可避免的过程，往往会导致运动功能下降，例如不稳定的步态、虚弱，甚至难以完成日常活动。骨骼肌的收缩和运动是由运动神经元通过神经肌肉接头（NMJ）控制的，这是运动神经元与肌肉纤维之间的突触连接。随着年龄增长，肌肉 ...

Duchenne muscular dystrophy is a severe and progressive disorder caused by mutations in the dystrophin (DMD) gene that lead to malfunction or absence of dystrophin. This protein stabilizes the ...

该研究表明Sirt6蛋白在老年小鼠肌肉中降低，在骨骼肌中敲除Sirt6导致抗肌萎缩蛋白Dystrophin蛋白水平降低，导致过早的神经肌肉接头（NMJ）退化变性 ...

Children born with the degenerative disease have a genetic mutation that stops them producing dystrophin, a protein that is vital for muscle strength and function. The same disorder also occurs in ...

Indicated for patients with a confirmed mutation of the dystrophin gene amenable to exon 51 skipping, the drug could be used in around 13% of the disease population. Sarepta’s share price almost ...

SRP-5051 is designed to bind to exon 51 of dystrophin pre-mRNA, resulting in exclusion of this exon during mRNA processing in patients with genetic mutations amenable to exon 51 skipping. With this ...

A Phase 3 clinical trial investigating Elevidys (delandistrogene moxeparvovec), a gene therapy for Duchenne muscular ...

Sarepta Therapeutics has ended development of a late-phase Duchenne muscular dystrophy (DMD) drug candidate over a safety problem. The safety signal deprived Sarepta of a near-term shot at accelerated ...

Duchenne muscular dystrophy is caused by pathogenic variants in the DMD gene that lead to a lack of functional dystrophin and muscle loss. Delandistrogene moxeparvovec delivers a gene that codes ...

I started calling myself an artist in 2019. And that is what I do, I make art. I make really good art. I even have a degree ...

US biotech Sarepta will continue to be responsible for clinical development and manufacturing of the drug SRP-9001, also known as (AAVrh74.MHCK7.micro-dystrophin),with global clinical development ...