TREAT-NMD Core Datasets

This is an example form to illustrate the dataset; neither the structure nor the wording is binding. Important features such as validation and plausibility checks are not implemented. This example is mainly intended as a baseline form; a follow-up form may need modifications to incorporate previously collected data.

Diagnosis

Live mapping

When did the individual receive the diagnosis?

Which method(s) were used to establish the initial diagnosis of DMD or BMD?

Genetic/molecular test

Physical examination

Muscle biopsy

Biomarkers

Was the diagnosis made as a result of screening?

Yes, family screening

Yes, newborn screening

Yes, prenatal screening

Does the individual have genetically proven DMD/BMD?

Yes, DMD

Yes, BMD

Pulmonary function

Live mapping

Non-invasive ventilation (example for patient-reported registries)

Have you ever used non-invasive ventilation due to your neuromuscular condition?

I am currently using non-invasive ventilation

I have previously used non-invasive ventilation, but am not currently

I have never used non-invasive ventilation

I don't know

Non-invasive ventilation (example for clinician-reported registries)

Is the individual currently using non-invasive ventilation due to their neuromuscular condition?

Yes

I don't know

Therapies and medication

Steroids

Live mapping

Have you ever taken steroids as treatment for your muscular dystrophy?

Yes, I am currently taking steroids

Yes, I have previously taken steroids

No, I have never taken steroids

Hospitalisations and comorbidities

Further medical conditions

Live mapping

Show form for follow-up with previous entry 18 months ago

Has the individual been diagnosed with any other illnesses or conditions alongside their muscular dystrophy that held in the last 12 months?

Yes

I don't know