Care and Trial Site Registry

Not applicable; available for all patients

Economic reasons

Not available in our region

Patients refuse

Other reasons (please specify below)

Manual wheelchair

Please specify "other reasons" for not receiving manual wheelchair here

Electric wheelchair

Please specify "other reasons" for not receiving electric wheelchair here

Regular physiotherapy

Please specify "other reasons" for not receiving regular physiotherapy here

Assisted ventilation
(non-invasive via mask or invasive via tracheostomy)

Please specify "other reasons" for not receiving home ventilation here

Scoliosis surgery

Please specify "other reasons" for not receiving scoliosis surgery here

Any comments:

	Yes	Most frequent reason
We don't recommend to attend the neuromuscular clinic at least once a year
Difficulty to provide an appointment / long waiting list at our clinic
Economic difficulties / travel is too expensive
Distance: clinic too far away / journey is too long
Patients prefer their general practitioner for regular assessment
Patients don't want to come so often
Others:

Neuromuscular doctor	minutes
Physiotherapist	minutes
Nurse	minutes

	2x per year	1x per year	< 1x per year	Not regularly / only with symptoms	Not performed
North Star Ambulatory Assessment
Functional abilities (e.g. time testing)
Muscle force (myometry or manual muscle testing)
Range of motion of joints
Echocardiography
Measurement of forced vital capacity (FVC)
DEXA or CT scan to measure bone density
Any comments:

	2x per year	1x per year	< 1x per year	Not regularly / only with symptoms	Not performed
Functional abilities (e.g. time testing)
Muscle force (myometry or manual muscle testing)
Range of motion of joints
Echocardiography
Measurement of forced vital capacity (FVC)
DEXA or CT Scan to measure bone density
Any comments:

	2x per year	1x per year	< 1x per year	Not regularly / only with symptoms	Not performed
Functional abilities (e.g. time testing)
Muscle force (myometry or manual muscle testing)
Range of motion of joints
Echocardiography
Measurement of forced vital capacity (FVC)
DEXA or CT Scan to measure bone density
Any comments:

My site

Patient cohort

Clinical trial infrastructure

Care settings

Research and education

Duchenne care

Please give a rough estimate of the percentage of your patients with DMD that come to your neuromuscular clinic at least once a year.

What are the reasons why some of your DMD patients come less than once a year to your clinic?

Please select all the reasons that apply. Then choose the most frequent reason and select it.

What is the typical length of time a patient with DMD spends with each of the following health care providers at each appointment in your clinic?

Please imagine a typical ambulatory boy with DMD at your clinic.
What kind of clinical assessments does an ambulatory child with DMD get? At what frequency are they usually carried out?

Please give an answer for each type of assessment.

Please imagine a typical non-ambulatory child with DMD without respiratory complications or cardiomyopathy at your clinic.
What kind of assessments does a non-ambulatory boy without respiratory complications or cardiomyopathy get? At what frequency are they usually carried out?

Please give an answer for each type of assessment.

What kind of clinical assessments does an adult with DMD get? At what frequency are they usually caried out?

Please give an answer for each type of assessment.

There are different approaches to the treatment of ambulant patients with DMD with corticosteroids. Which of the following is closest to yours?

Please select the most important reason for your decision to start a treatment with corticosteroids.

Which of the following approaches concerning the ending of the treatment with corticosteroids is closest to yours?

Please select the most appropriate answer.

Please give a rough estimate of the percentage of your ambulant DMD patients receiving corticosteroids, provided that it is indicated.

Please give a rought estimate of the percentage of your non-ambulant DMD patients receiving corticosteroids.

What measures do you recommend on a regular basis to recognize or prevent side effects of corticosteroid treatment?

Please give an answer for each of the 8 measures.

Is the following equipment/treatment available for all of your patients, when it is indicated?

Please choose one answer for each item.

If not all patients receive the mentioned equipment/treatments although they are indicated (see previous question) what is the most important reason for this?

Please choose one reason for each of the 5 items.

Are you interested in attending specific training courses about treatment in DMD?

Please check each topic for its relevance to you.

Are you aware of the Family Guide "The diagnosis and management of Duchenne Muscular Dystrophy" created by TREAT-NMD and the patient organizations PPMD, UPPMD and the MDA?

	Corticosteroids are offered to all patients
	Corticosteroids are only prescribed upon request
	Corticosteroids use is not offered
Other approaches / comments:

Patient's age:		Please specify the age:
Stage of disease:
		Ideal phase to start: Making motor progress
		Plateau phase
		Motor decline
Not applicable:
Other reasons / comments:

	I usually stop when the patient has lost ambulation
	I usually continue after loss of ambulation but I generally stop if side effects don't allow continuation of treatment
	Not applicable because steroids are not used routinely
Other approaches / comments:

	Yes, regularly recommended	Yes, but irregularly recommended	No, not recommended
Completion of vaccination before start of steroids
Regular measurements of weight and height
Contact with a dietician
Ophthalmologic control for cataracts
Control of blood glucose level or glucosuria
Blood pressure measurement
Control of vitamin D levels
Control of bone mineral density
Any comments:

	Yes, for all patients	Yes, for most patients	Yes, for a few patients	None of my patients
Manual wheelchair
Electric wheelchair
Regular physiotherapy
Assisted ventilation (non-invasive via mask or invasive via tracheostomy)
Scoliosis surgery
Any comments:

	Yes, very important	Yes, less important	No, not necessary
Psychosocial care
Cardiac management
Pulmonary management
Neuromuscular management
Corticosteroid therapy
Orthopaedic management
Gastrointestinal management
Bone health
Palliative care
Others

My site

Patient cohort

Clinical trial infrastructure

Care settings

Research and education

Duchenne care

Please give a rough estimate of the percentage of your patients with DMD that come to your neuromuscular clinic at least once a year.

What are the reasons why some of your DMD patients come less than once a year to your clinic?

Please select all the reasons that apply. Then choose the most frequent reason and select it.

What is the typical length of time a patient with DMD spends with each of the following health care providers at each appointment in your clinic?

Please imagine a typical ambulatory boy with DMD at your clinic. What kind of clinical assessments does an ambulatory child with DMD get? At what frequency are they usually carried out?

Please give an answer for each type of assessment.

Please imagine a typical non-ambulatory child with DMD without respiratory complications or cardiomyopathy at your clinic. What kind of assessments does a non-ambulatory boy without respiratory complications or cardiomyopathy get? At what frequency are they usually carried out?

Please give an answer for each type of assessment.

What kind of clinical assessments does an adult with DMD get? At what frequency are they usually caried out?

Please give an answer for each type of assessment.

There are different approaches to the treatment of ambulant patients with DMD with corticosteroids. Which of the following is closest to yours?

Please select the most important reason for your decision to start a treatment with corticosteroids.

Which of the following approaches concerning the ending of the treatment with corticosteroids is closest to yours?

Please select the most appropriate answer.

Please give a rough estimate of the percentage of your ambulant DMD patients receiving corticosteroids, provided that it is indicated.

Please give a rought estimate of the percentage of your non-ambulant DMD patients receiving corticosteroids.

What measures do you recommend on a regular basis to recognize or prevent side effects of corticosteroid treatment?

Please give an answer for each of the 8 measures.

Is the following equipment/treatment available for all of your patients, when it is indicated?

Please choose one answer for each item.

If not all patients receive the mentioned equipment/treatments although they are indicated (see previous question) what is the most important reason for this?

Please choose one reason for each of the 5 items.

Are you interested in attending specific training courses about treatment in DMD?

Please check each topic for its relevance to you.

Are you aware of the Family Guide "The diagnosis and management of Duchenne Muscular Dystrophy" created by TREAT-NMD and the patient organizations PPMD, UPPMD and the MDA?

Please imagine a typical ambulatory boy with DMD at your clinic.
What kind of clinical assessments does an ambulatory child with DMD get? At what frequency are they usually carried out?

Please imagine a typical non-ambulatory child with DMD without respiratory complications or cardiomyopathy at your clinic.
What kind of assessments does a non-ambulatory boy without respiratory complications or cardiomyopathy get? At what frequency are they usually carried out?