Beyond DYNE-251, the company is expanding its DMD franchise with preclinical programmes aiming at other exons such as 44, 45, and 53. "EC grants orphan drug designation to Dyne Therapeutics’ DMD ...

The large DMD gene has 79 exons. For DMD patients with suitable mutations, exon skipping is one way to convert out-of-frame mRNA translation to a properly aligned reading frame. Oligonucleotides ...

In addition to DYNE-251, Dyne is building a global DMD franchise and has preclinical programs targeting other exons, including 53, 45 and 44.?? DMD is a rare disease caused by mutations in the gene ...

In addition to DYNE-251, Dyne is building a global DMD franchise and has preclinical programs targeting other exons, including 53, 45 and 44. DMD is a rare disease caused by mutations in ...

The cost savings will allow the firm to hire new employees who can support clinical trials of its DMD candidates and advance other therapeutic programs.

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 ...