European Registry of Patients With Infantile-onset Spinal Muscular Atrophy

European Registry of Patients With Infantile-onset Spinal Muscular Atrophy

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.

Inclusion Criteria:

• Spinal Muscular Atrophy diagnosed in childhood (before 18 months) and genetically confirmed.
• For patients with SMA type 1: Never acquired independent sitting position (more than 30 seconds, without hand support or any external support)
• For patients with SMA type 2 or 3: Patient treated with a market approved treatment for SMA or with a treatment in an expanded access program
• Any age
• Patients over 18 years of age or parent(s)/legal guardian(s) of patients < 18 years of age not opposed to data collection for research purposes

Exclusion Criteria:

• None