Update on Sarepta’s DMD gene therapy trial

Published Date
26/03/2019
Author
Beatriz Bustillo Ramirez
Category
Research
Orange box stating: Breaking news research

Sarepta Therapeutics has provided an update on the four boys participating in its gene therapy trial for Duchenne muscular dystrophy. It’s now been nine months since the boys were treated. They are all doing well and no serious safety concerns have been reported. Tests have also shown that their creatine kinase levels have decreased and their muscle function appears to be better than what is usually seen in Duchenne patients of the same age. Although the boys will continue to be followed up for 36 months in total, these results are promising and the company is now recruiting for a Phase II trial in USA.

For more information, access the conference call slides and audio here.

If you have any questions about this news story or any other DMD research, please contact the MDUK Research Line on 020 7803 4813 or email research@musculardystrophyuk.org.

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