Preview of the questionnaire
Personal data
What is your NHS number?
Your NHS number is a 10 digit number, like 485 777 3456. It is on any letter you receive from the NHS.
Please select the neuromuscular centre from which you receive your main care for DMD, from the list below.
Diagnosis
The genetic report is a document that includes information about your diagnosis. If you have the genetic report yourself please click on the following link to upload the document:
Upload genetic report (link opens in new window)
If you are unable to upload the report, you can send a photocopy by post to us.
What is your genetically confirmed diagnosis?
This information will be found on any genetic report that you will have received.
Do you have a copy of your genetic report(s)?
If you consent, we will contact the clinic selected in the section “Personal data” directly to request a copy of your genetic report.
Motor function
Which of the following activities are you currently able to carry out?
Do you currently use mechanical ventilation equipment?
Wheelchair use
Do you currently use a wheelchair or other mobility device (e.g. buggy/pushchair or electric scooter)?
Medication
Are you currently taking oral corticosteroids (steroids) to manage your DMD symptoms?
Have you ever taken any disease-modifying therapy for your DMD?
These are therapies that specifically aim to improve muscle strength or slow down the disease progression. In the UK the only approved disease-modifying therapy is Translarna (ataluren), but you might receive other medication through compassionate use or other early access programmes, for example Exondys 51 (eteplirsen), Vyondys 53 (golodirsen) or Amondys 45 (casimersen).
If yes, what medication have you taken?
If you have taken more than one medication, click on the button “Add another medication” to add further details.
Have you taken any over the counter or non prescribed medication(s) or supplement(s) in the last 12 months?
Please only count those drugs or supplements you have taken for at least 14 days in a row.
Are you currently taking testosterone (e.g. Sustanon)?
Participation
Have you ever participated in a DMD-related clinical trial?
Please consider any interventional and natural history studies. Information about DMD-related research studies or clinical trials can be found on the DMD Hub website (link opens in new window).
If yes, which trial(s) are you participating in or have you participated in?
To add multiple trials, click on the button “Add another clinical trial”.
How important would the following criteria be for your decision to participate in an interventional study?
An interventional study is a research study performed on people that is aimed at evaluating a medical, surgical, or behavioral intervention.
Would you be willing to participate in an interventional study requiring the following tests?
How far would you be willing to travel to another site to participate in an interventional study?
This question refers to the distance from the address you specified during registration. You can view and edit it on the page Contact data (link opens in new window).
How important would the following criteria be for your decision to participate in a natural history study?
A natural history study collects health information over time to understand how the medical condition or disease develops and to give insight into how it might be treated.
In case you are not intereseted in participating in a natural history study, just leave the option “Not specified” selected.
Would you be willing to participate in an natural history study requiring the following tests?
How far would you be willing to travel to another site to participate in a natural history study?
Which kinds of trials would you like to be contacted about?
This is a medical field which focuses on the genetic modification of cells to produce a therapeutic effect or the treatment of disease by repairing or reconstructing defective genetic material.
There are currently two types of mutation-specific therapies in clinical trials for Duchenne: nonsense mutation suppression (individuals with Duchenne who have a nonsense mutation may be candidates for nonsense mutation read-through) and exon skipping (individuals with Duchenne who have certain “out-of-frame” deletions may be candidates for exon skipping).
These are research studies performed on people that are aimed at evaluating a medical, surgical, or behavioral intervention.
A study collecting health information over time to understand how the medical condition or disease develops and to give insight into how it might be treated.