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. 2021 Mar 9;44(3):566–592. doi: 10.1002/jimd.12370

TABLE 1.

Outcome parameters and their ratings of importance

Outcome parameter Median rating of importance of outcome
Survival 9
Health‐related quality of life 9
Metabolic stability 8
Cognitive development 8
Epilepsy 8
Metabolic stroke 7
Vision and hearing 7
Early diagnosis 7
Cardiomyopathy 7
Kidney dysfunction 7
Pancreatitis 6
Normal growth 6
Neutropenia 6
Anaemia 5.5
Bone health 5

Note: Each outcome parameter was rated by the panellists and the patient representatives from 0 (lowest importance) to 9 (most critical for decision‐making). The second column represents the median value of all the ratings.