Disease-modifying therapies (DMT)

Registries should use the general consensus of what constitutes a DMT for a given disease area and include predefined values in their data collection form. Where no such consensus exists, registries should define a DMT as a pharmacological treatment that affects the underlying pathology or pathophysiology of the disorder (cf. Cummings 2006). When in doubt, a given treatment should be considered as disease-modifying.

Items in this group
DMT received CR  PR  datestamped 

Specifies whether the individual has ever received a disease-modifying therapy for their neuromuscular condition.

Item type: single selection
Value ID Description
Yes

The individual has received an approved disease-modifying therapy

No

The individual has never received a disease-modifying therapy, even though there exists an approved DMT for their neuromuscular condition (regardless of whether the DMT is approved in the individual's country and whether they are eligible or not)

Not applicable

There exists no approved disease-modifying therapy for the individual's neuromuscular condition

Record

DMT episode CR          episode   

DMT CR 

International Nonproprietary Name (INN) of the disease-modifying therapy the individual is receiving or has received at some point.

Item type: free text
DMT status CR 

Specifies whether this therapy is ongoing or stopped.

Item type: single selection
Consistency rules:

If a value for Stop date of this record is provided, this item must have the value Stopped.

Usage in other datasets: SMADMD
Value ID Description
Ongoing

The individual has been receiving the therapy as of the date specified in Ongoing date and has been continuously receiving it from the date specified in Start date on (if specified)

Stopped

The individual has stopped continuously receiving the therapy since the date specified in Start date