On the 31st of January, a group of patients, patient organizations, clinicians, researchers and companies developing drugs for Duchenne came together for a workshop to discuss and evaluate the outcome measures that are currently used in clinical trials.
One of the sessions during the day involved a discussion on muscle biopsies. This session was led by the muscle biopsy working group which includes a patient, a representative from a patient organization, clinicians and researchers. During the discussions in that session it was felt that very little data had been collected on the patient perspective on muscle biopsies.
To address this the muscle biopsy working group decided to devise this questionnaire to allow us to better understand your views and look at how we can improve the patient and caregiver experience when carrying out muscle biopsies in clinical trials.
Please only complete the questionnaire if the child in your care has taken part in a clinical trial requiring an open muscle biospy. We want to know your feelings on open muscle biopsies (so not needle biopsies or skin biopsies). An open muscle biopsy is a surgical procedure where an incision is made in the skin and subcutaneous fat to reach the muscle and remove a part of it for analysis procedures.
We hope you will fill out this questionnaire to help us collect this important information.
There are three parts to this questionnaire. Parts 1 and 3 are to be completed by the caregiver, and Part 2 by the child with help from the caregiver.