The genetic information on DNA is transcribed into messenger RNA (mRNA) and translated to the amino acid sequence by transfer RNA (tRNA) on the ribosome. Modified nucleosides within RNA are involved in maintaining and regulating the protein synthesis system.
Sarepta’s confirmatory Duchenne trial fails to meet endpoint, complicating future for controversial drug
Sarepta and Roche’s confirmatory study for a Duchenne gene therapy failed to hit its primary endpoint, complicating plans to convert the drug’s label into a