| Key inclusion criteria |
- Genetic documentation of 5q SMA (a homozygous deletion, mutation, or compound heterozygote in SMN1) with symptom onset after 6 months of age
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- The presence of the following features at screening:
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- Able to sit independently but never able to walk independently (defined as the ability to walk ≥15 ft unaided)
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- Estimated life expectancy more than two years from screening
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| Key exclusion criteria |
- Respiratory insufficiency (ie, receipt of invasive or noninvasive ventilation for >6 hours during a 24-hour period)
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- Need for a gastric tube placed for a majority of feeds to provide adequate nutrition
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- Presence of severe contracture (ie, any contracture that could interfere with assessment of the HFMSE score, according to the investigator)
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- Evidence of severe scoliosis on radiography (ie, spine curvature with a Cobb angle of >40 degrees)
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- Any ongoing medical condition that would interfere with the conduct and assessments of the study (eg, medical disability such as wasting or cachexia, severe anemia)
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