LGMD 2E gene therapy trial starts in the USA

Published Date
08/11/2018
Author
Jenny Sharpe
Category
Research
Orange box stating: Breaking news research

Myonexus Therapeutics has initiated the first-ever gene therapy (MYO-101) trial for people with limb girdle muscular dystrophy type 2E (LGMD 2E). The trial is assessing two doses of the gene therapy in children with LGMD 2E between the ages of four and fifteen years old.

The first child in the low-dose group was treated in October and the remaining participants for that group are expected to be treated by the end of 2018. Preliminary data is expected by early 2019.

For more information, please read Myonexus Therapeutics’ press release.

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

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