DMD, a genetic disorder affecting 1 in 5000 male births, is caused by mutations in the DMD gene. While it primarily leads to muscle ... is affected late in its maturation. Using RNA sequencing and ...

Antisense oligonucleotides to the boundary sequences of exon and intron 23 of the dystrophin gene in the mouse model of DMD (mdx) have previously been shown to result in successful splicing-out of the ...

Exon skipping is one approach to treat DMD that involves using small, synthetic DNA molecules called antisense ... stop codon into a patient’s mRNA sequence, causing the production of truncated ...

A new paper published in Gene Therapy raises serious concerns about the effectiveness of gene therapy for Duchenne muscular ...

Two dose levels of a single-administration gene therapy were well-tolerated and led to functional improvements in ambulatory ...

Sarepta Therapeutics announced that a patient with Duchenne muscular dystrophy (DMD) who received Elevidys (delandistrogene moxeparvovec-rokl) died following treatment. Elevidys is administered as ...

New data on Sarepta's gene therapy for Duchenne muscular dystrophy (DMD) has gone a long way towards building confidence in its efficacy – but have introduced a concern about its safety. Updated ...

Two-year data showed significant functional improvements in patients treated with delandistrogene moxeparvovec compared to controls, despite initial trial endpoint failure. The treated group ...

RGX-202, aims to address the root cause of DMD by delivering a functional copy of the gene that encodes microdystrophin, a protein that is missing or defective in DMD patients. DMD is a form of ...

DMD, a genetic disorder affecting 1 in 5000 male births, is caused by mutations in the DMD gene. While it primarily ... Using RNA sequencing and functional brain analyses the team of 21 scientists ...