From: SMArtCARE - A platform to collect real-life outcome data of patients with spinal muscular atrophy
Patient visits (days) | ||||||
---|---|---|---|---|---|---|
1 | 14a | 28a | 63a | 180 | Every 6 months (every 4 monthsa) | |
Baseline data including genetic test results | X | |||||
Current medical history and clinical examination - including motor milestones in children < 12 years of age | X | X | X | X | X | X |
Administration of nusinersen a | X | X | X | X | X | X |
Physiotherapeutic assessments: CHOP INTEND • All children < 2 years of age • All patients > 2 years of age without ability to sit HFMSE • All patients > 2 years of age with ability to sit • If CHOP INTEND score > 50: CHOP INTEND and HFMSE • If CHOP INTEND score > 60: HFMSE instead of CHOP INTEND RULM • All patients > 2 years of age with ability to sit (in a wheelchair) 6-MWT • All ambulant patients > 3 years of age | X | X | X | X | ||
ALS Functional Rating Scale (in adult patients) | X | X | X | X | ||
Pulmonary function b | X | X | X | |||
Documentation of adverse events | Ongoing |