Table 5.
Baseline data including genetic test results |
Current medical history and clinical examination o including motor milestones in children < 12 years of age |
Physiotherapeutic assessments: CHOP INTEND o All children < 2 years of age o All patients > 2 years of age without ability to sit |
HFMSE o All patients > 2 years of age with ability to sit o If CHOP INTEND score > 50: CHOP INTEND and HFMSE o If CHOP INTEND score > 60: HFMSE instead of CHOP INTEND |
RULM o All patients > 2 years of age with ability to sit (in a wheelchair) |
6MWT o All ambulant patients > 3 years of age |
ALS Functional Rating Scale (in adult patients) |
Pulmonary functiona |
Documentation of adverse events |
Note: aIf the patient is sufficiently cooperative due to age.