Furthermore, impressive results in the DMD program, particularly in exon skipping and dystrophin expression, exceeded expectations, positioning Avidity Biosciences favorably for regulatory discussions ...

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 ...

Regulating drug discovery and pharmaceutical manufacturing, the FDA ensures the safety and effectiveness of innovative ...

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

The US biopharma will discontinue development of the Exondys 51 (eteplirsen) follow up treatment to focus on other treatment ...

Elevidys sales have increased since the FDA made a controversial choice to expand the therapy's use. Meanwhile, Sarepta is ...

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 ...

Specific mutations in the DMD gene influence how long patients retain the ability to walk, even when treated with ...

Duchenne, has been a transformative force in the fight against Duchenne muscular dystrophy (DMD). Following her son Hawken’s ...

Sales of Elevidys are quickly gaining ground on Sarepta's exon-skipping trio, which need to be dosed chronically. In the ...

Presented additional data from the Phase 1 clinical trial ENTR-601-44-101 and new preclinical data supporting ENTR-601-45 for DMD at 2024 World Muscle Society Annual Congress – – On track to submit gl ...