NS Pharma’s Viltepso (viltolarsen) has been approved in patients who have confirmed mutation of the DMD gene that is amenable to exon 53 skipping. The DMD gene is made up of 79 exons ...

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 ...

Around 13% of DMD patients respond to exon 51 skipping, but PepGen reckons other drugs in its pipeline will address mutations that are central to around 35% of all cases of the disease.

Of note, exon-skipping oligonucleotides and gene therapy in DMD have been approved under the FDA's accelerated approval ...

“To me, that's quite compelling.” As for exon-skipping therapies, another new-generation option for DMD, he noted that “these ...

The paper, by Y.-L. Min at University of Texas Southwestern Medical Center in Dallas, TX, and colleagues was titled, "CRISPR-Cas9 corrects Duchenne muscular dystrophy exon 44 deletion mutations in ...

The company intends to develop the drug as a potential treatment for DMD patients who are exon 44 skipping amenable. Entrada is also evaluating multiple other DMD candidates in preclinical studies ...

Scientists have been searching for ways to cure DMD for as long as they have known about the disease. Today, based on the wealth of knowledge about the underlying genetic and molecular mechanisms ...

in patients who are exon 50 skipping amenable.” The posters will be presented during Poster Session 2 (DMD Treatments, 5:15-6:15 pm CET) and will be available on the Scientific Presentations ...

New Gene Therapy Approach Shows Promise for Duchenne Muscular Dystrophy July 24, 2024 — Researchers have made a significant breakthrough in developing a new gene therapy approach that restores ...

We identified a novel and significant association between Duchenne muscular dystrophy and autism spectrum disorder/pervasive developmental disorder. The eight cases with both diagnoses presented ...

Zinc supplementation showed limited benefits in improving muscle health and hydration in boys with DMD. Nearly 30% of participants had low zinc levels initially, with slight improvement post ...