The colorful threads in this image are muscle fibers. This spotted rainbow illustrates a potential gene therapy approach to treating Duchenne muscular dystrophy (DMD) – a rare, debilitating, and fatal ...

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 ...

Duchenne muscular dystrophy results from mutations in the DMD gene that encodes the dystrophin protein. There are many types of mutations that can cause the disease; each disrupts the reading frame ...

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 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 ...

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).

Duchenne muscular dystrophy (DMD) is a rare genetic disorder that is characterised by progressive muscle degeneration and weakness, and is caused by mutations in the dystrophin gene. Currently ...