Astellas Pharmaceuticals has announced that the first child has now received treatment in its new gene therapy clinical trial, called VALOR, for X-linked myotubular myopathy (XLMTM).
First patient receives treatment in new gene therapy trial for XLMTM
5 May 2026
Currently only recruiting in the USA and Canada, the VALOR study is an early-phase (phase 1/2) clinical trial testing an investigational gene therapy known as ASP2957. It is designed for boys under the age of three living with XLMTM and will assess both safety and how well the treatment works.
This marks an important step forward, as the trial moves from laboratory research into clinical testing following review by regulatory authorities. Researchers will now begin to learn more about how this potential therapy works in the body.
As with all early-stage clinical trials, safety is the top priority and participants in the VALOR study will be closely monitored throughout.
While it is still early days for the VALOR trial, this development represents a new stage in efforts to better understand and potentially treat XLMTM.
