The findings support the clinical benefits of delandistrogene moxeparvovec in patients with Duchenne muscular dystrophy (DMD) ...

Participants with Duchenne muscular dystrophy (DMD) who received RGX-202 demonstrated functional improvement and better ...

Prime editing is an advanced gene editing technology that enables precise DNA corrections without introducing double-stranded ...

PBGENE-DMD is the first in vivo gene editing program that has the potential to transform the treatment paradigm and deliver durable functional improvement for most patients, as up to 60% of those ...

Scientists present current evidence for a new gene therapy for Duchenne muscular dystrophy called delandistrogene moxeparvovec. AAN Evidence in Focus articles highlight the strength of the current ...

The molecular diagnosis of DMD was established using MLPA at the National Research ... The deletion included the PARK2 gene but the exact location of the breakpoints was unknown. The boy's father did ...

Sarepta Therapeutics said it will update its prescribing information for Elevidys® (delandistrogene moxeparvovec-rokl) after acknowledging the sudden death of a patient with Duchenne muscular ...

The givinostat approvals in the US and UK, along with the promising long-term data we are generating, bring us closer to our goal: ensuring that every patient, regardless of location, has access ...

Gene therapies have set a high bar for outcomes in pharma by delivering curative treatments for difficult diseases in a one-and-done punch. But for Duchenne muscular dystrophy, the results of ...

It is an X-linked recessive disorder that occurs due to changes in the DMD gene, which codes for the protein dystrophin. Dystrophin is a protein that plays a role in strengthening muscle fibers ...

Genetic profiling has revealed significant changes in gene expression in DMD. These changes offer insights into the ... The N-terminus is the location of one of the two, whereas the C-terminus is the ...

regardless of the specific mutation location on the DMD gene. Preclinical data showed that when delivered through an under-the-skin (subcutaneous) injection using the company’s proprietary ...