DMD+Results

Results:

Exon skipping manipulates the splicing mechanism and causes the target exon to be skipped, restoring the reading frame and allowing for translation. = =

=
=====

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5338848/

Success of Eteplirsen clinical trials?


 * Following initial treatment of Eteplirsen all patients were confirmed to have successful exon skipping and produced a shortened PCR product.


 * Western Blot Assay detects increasing presence of dystrophin protein at successive monitoring points in the longitudinal studies beginning at week 24 of treatment.


 * In one study by the FDA, production of dystrophin had increased by 11 fold by week 180 and detection of dystrophin fibers by 18 fold.


 * Results indicate that there was no adverse reaction to the injection of the exon skipping inducers and that muscles in close proximity to the injection sites were found to be producing up to ~42% of dystrophin as found in healthy muscle.

Materials and Methods (Back)
= =