Duchenne muscular dystrophy is a severe and progressive disorder caused by mutations in the dystrophin (DMD) gene that lead ...

The European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) has once again snubbed Translarna ...

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

Sidra Medicine s Genetic and Genomic Medicine clinic is providing life altering gene therapy for rare genetic disorders. In a ...

While Dyne Therapeutics Inc.’s DYNE-251 demonstrated high levels of dystrophin expression and functional improvement in boys with Duchenne muscular dystrophy who are amenable to exon 51 skipping, ...

SAVANNAH, Georgia — Heart problems are common in the vast majority of neuromuscular disorders, and cardiac monitoring of ...

The improvement in dystrophin was approximately in line with the 5.9% unadjusted improvement seen with Viltepso in a phase 2 study that supported its 2020 approval by the FDA. Shares in Wave rose ...

圣地亚哥 - Capricor Therapeutics (NASDAQ: CAPR) 已开始向美国食品和药物管理局(FDA)滚动提交生物制品许可申请(BLA),申请其产品候选药物deramiocel用于治疗杜氏肌营养不良(DMD)患者的心肌病。该公司预计将在2024年底前完成提交程序,并希望该申请能够获得优先审查资格,这可能会加快批准时间。 Deramiocel (CAP-1002)由异体心肌球 ...

Elevidys (delandistrogene moxeparvovec) is a gene that produces a short protein that resembles dystrophin. It is injected in one IV infusion. There is no cure for DMD. This inherited condition is ...

SAVANNAH, Ga. -- Delandistrogene moxeparvovec (Elevidys), a gene therapy approved to treat Duchenne muscular dystrophy, ...

While success is far from guaranteed in bio-land, with this week’s raising, Percheron has the horsepower to have a decent ...

Additional positive data from the Company’s completed Phase 1 clinical trial evaluating ENTR-601-44 reinforces its safety profile and supports ...