Table 4 Top 5 most frequently reported complicating issues to choose 'YES' or 'NO' for HSCT

1. Developmental delay¹

2. Older age

3. Cardiomyopathy

4. ERT prior to HSCT desirable

5. Uncertain (CNS) prognosis

Complicating for 'NO' for HSCT

1. ERT prior to HSCT might improve symptoms

2. Developmental delay¹

3. Long-term course unknown

4. Unknown role for HSCT in milder phenotype

5. Early diagnosis