Entrada plans to initiate clinical testing of ENTR-601-44 in Duchenne MD patients amenable to exon 44 skipping later this year.

While the last decade has brought considerable progress for patients with DMD, substantial unmet need remains. Several ...

Dystrophin is a protein that plays a role in ... Research highlights that the DMD gene is the largest known human gene, featuring 79 exons (coding regions). The gene’s large size means it ...

Three of its drugs skip exons — or specific places in the genome — to help the body make dystrophin. Its gene therapy, ...

The DMD gene is made up of 79 exons, and mutations in that code can result in a deficiency in dystrophin which is responsible for the muscle wasting in DMD. Exon-skipping drugs are used to patch ...