News and press releases about the Joshua Frase Foundation and its efforts behind CNM/MTM

Fri, Sep 17, 2021

The biggest questions facing gene therapy, the XLMTM community, and Astellas after fourth patient death

After three patients died last year in an Astellas gene therapy trial, the company halted the study and began figuring out how to safely get the program back on track. They would, executives eventually explained, cut the dose by more than half and institute a battery of other measures to try to prevent the same thing from happening again.