Clinical Trials Arena on MSN15d
Avidity’s del-zota elicits 25% increase in dystrophin in DMD patients
The company plans to submit a biologics licence application to the US Food and Drug Administration later this year.
Clinical Trials Arena on MSN16d
Dyne’s DMD trial advances patient mobility and dystrophin expression
The Phase I/II Deliver trial sought to establish the impact of DYNE-251 on several mobility-related endpoints, seeing an increase over baseline at 12 months.
BioWorld6d
PBGENE-DMD restores dystrophin functioning in DMD
Precision Biosciences Inc. recently presented a new gene-editing approach, PBGENE-DMD, which could allow life-long benefits to patients with Duchenne muscular dystrophy (DMD).
The American Journal of Managed Care12d
Delandistrogene Moxeparvovec Shows Long-Term Impacts at 3 and 5 Years in DMD
Study results support the role of functional dystrophin and suggest that delandistrogene moxeparvovec stabilizes or slows ...
Precision Medicine Online14d
Patient Dies After Treatment With Sarepta's Duchenne Muscular Dystrophy Gene Therapy
The firm's stock price dropped 27 percent on Tuesday following the news that the young man died from acute liver failure after getting Elevidys.
BioWorld7d
EDG-4131 active in model of Becker muscular dystrophy
Researchers from Edgewise Therapeutics Inc. have previously developed and characterized a novel bmx mouse model of Becker muscular dystrophy (BMD), which harbors a deletion of murine Dystrophin exons ...
Yahoo Finance15d
Avidity’s del-zota elicits 25% increase in dystrophin in DMD patients
Avidity Biosciences’ Phase I/II trial of ribonucleic acid (RNA) therapy, del-zota, has increased dystrophin in patients with Duchenne muscular dystrophy (DMD). Results from the company’s ...