Saves the questions and what to expect into your notes, next to the visit they belong to.
Keep this study
Lead
Astellas Gene Therapies
Age
Any age
Sex
MALE
Healthy volunteers
Not accepted
You may be eligible if
Patient diagnosed with XLMTM resulting from a confirmed mutation in the MTM1 gene, or a combination of XLMTM genetically confirmed family history and muscle biopsy
Patient is male
Access to available medical records for each patient
Signed informed consent by the parent(s) or legal guardians and/or assent by the patient (when applicable), unless the associated IRB provides an appropriate consent waiver
You may not be if
Patient data after participation in an interventional study designed to treat XLMTM (patient data prior to participation in an interventional study may be included)
