It is debatable, however, whether this creates a problem for society, because even though the frequency of the lethal mutations would increase, the number of babies born with DMD would decrease.

Duchenne muscular dystrophy (DMD) is the most common type ... It was the first targeted treatment for this type of mutation and has been shown to help increase the production of dystrophin.

Some types of muscular dystrophy typically affect ... It's only prescribed to people who have a confirmed mutation in the DMD gene. It delivers a protein to muscles in the skeleton, heart, and ...

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 ...

The US Food and Drug Administration (FDA) has granted Rare Pediatric Disease (RPD) designation to NS-051/NCNP-04, which is ...

The funding will be used to continue the clinical development of lead oligonucleotide PGN-EDO51 for DMD, as well as two other oligo drugs PGN-EDODM1 for myotonic dystrophy type 1 (DM1 ...

The phase 3 EMBARK trial has been evaluating the gene therapy in ambulatory boys aged four to seven years with a confirmed mutation in the DMD gene. Estimated to affect one in every 5,000 male births ...

Characterized by progressive muscle degeneration, this X-linked disorder results from mutations in the DMD gene ... tyrosine phosphatase non-receptor type 1 (PTPN1) and type 2 (PTPN2).

Unlike gene therapies, K884 targets DMD-affected cells regardless of the mutation, promoting functional muscle development. The breakthrough could improve muscle function and quality of life for all ...

DMD, a severe genetic disorder that causes muscle weakness and shortens lifespans, arises from mutations in the gene encoding dystrophin. While current treatments can extend patients' lifespans ...

Wave Life Sciences develops stereopure oligonucleotides targeting genetic mutations, with lead assets ... data from lead asset N531 in a rare type of DMD, which accounts for some 8-10% of the ...