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Table 2 Overview of rare disease with one disease-specific disease severity scale

From: Neutral theory: applicability and neutrality of clinical study endpoints where a disease-specific instrument is available

Disease

Disease-specific DSS

Number of indicators

Achalasia

Eckardt Score

40

Behcet’s syndrome

Krause's Behçet's Disease Activity Assessment

38

Creutzfeldt-Jakob disease

Creutzfeldt-Jakob Disease Neurological Status Scale

29

Fournier’s gangrene

Fournier’s Gangrene Severity Index

20

Guillain-Barré syndrome

Guillain-Barré Rating Scale

20

Atypical hemolytic uremic syndrome

Pediatric Neurologic Assessment Score for Hemolytic Uremic Syndrome

28

Palmoplantar psoriasis

Palmoplantar Psoriasis Area and Severity Index

3

Prader-Willi syndrome

Prader-Willi Syndrome Behavioral Questionnaire

39

Systemic lupus erythematosus

Lupus Severity Index

48

Systemic sclerosis

Modified Rodnan Skin Score

38