IO-SMA-Registry is a prospective, longitudinal and observational study which objective is to collect prospectively information on longevity, psychomotor development and respiratory function of patients with infantile-onset spinal muscular atrophy.
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Change from Baseline in survival
Timeframe: Baseline and then every 6 months until the end of the study, up to 5 years
Change from Baseline in psychomotor development
Timeframe: Baseline and then every 6 months until the end of the study, up to 5 years
Change from Baseline in the number of lower track infections
Timeframe: Baseline and then every 6 months until the end of the study, up to 5 years
Change from Baseline in ventilation use
Timeframe: Baseline and then every 6 months until the end of the study, up to 5 years
Change from Baseline in cough assist use
Timeframe: Baseline and then every 6 months until the end of the study, up to 5 years
Change from Baseline in Forced Vital Capacity
Timeframe: Baseline and then every 6 months until the end of the study, up to 5 years
Change from Baseline in diurnal saturation
Timeframe: Baseline and then every 6 months until the end of the study, up to 5 years
Change from Baseline in nocturnal hypercapnia
Timeframe: Baseline and then every 6 months until the end of the study, up to 5 years