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Table 1 Recommendations for the evaluation of patients with SMA

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
  1. aAdditional recommendations under treatment with nusinersen
  2. bIf the patient is sufficiently cooperative due to age