TABLE 1.
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.