$3.2 million per dose Elevidys fails to meet primary endpoint in phase 3 trial
A Phase 3 clinical trial investigating Elevidys (delandistrogene moxeparvovec), a gene therapy for Duchenne muscular ...
Investor's Business Daily on MSN24d
Avidity Biosciences, Up 441%, Nears Another Record High As Dystrophy Space Heats Up
In this condition, patients' bodies don't make enough for the dystrophin protein necessary to keep muscles intact. Sarepta ...
BioPharma Dive4d
Sarepta scraps a Duchenne drug as gene therapy sales rise
Elevidys sales have increased since the FDA made a controversial choice to expand the therapy's use. Meanwhile, Sarepta is ...
pharmaphorum4d
Sarepta pulls plug on Duchenne exon-skipping drug
Sales of Elevidys are quickly gaining ground on Sarepta's exon-skipping trio, which need to be dosed chronically. In the ...
BioWorld1mon
ENTR-601-45 restores dystrophin in Duchenne muscular dystrophy models
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 ...
Pharmabiz3d
US FDA grants orphan drug & rare paediatric disease designations to Cumberland Pharma’s Ifetroban to treat cardiomyopathy associated with DMD
Nashville, Tennessee Friday, November 8, 2024, 13:00 Hrs [IST] ...
FierceBiotech4d
Sarepta axes late-phase DMD drug over safety signal, FDA talks
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 ...
Business Insider22h
Avidity Biosciences’ Clinical Advancements Positioning for Regulatory Success
Furthermore, impressive results in the DMD program, particularly in exon skipping and dystrophin expression, exceeded expectations, positioning Avidity Biosciences favorably for regulatory discussions ...
Debra Miller
Duchenne, has been a transformative force in the fight against Duchenne muscular dystrophy (DMD). Following her son Hawken’s ...
biopharma-reporter4d
Sarepta discontinues development of Duchenne ASO treatment vesleteplirsen
The US biopharma will discontinue development of the Exondys 51 (eteplirsen) follow up treatment to focus on other treatment ...
腾讯网16d
Adv Sci：浙大团队揭示老年运动功能减退新机制，补充NMN可有效缓解
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 ...
Entrada Therapeutics Reports Third Quarter 2024 Financial Results
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 ...