A Real-World Study of Clinical Use of Available Treatments for Pediatric Patients With Spinal Muscular Atrophy

Real-World Evidence of Clinical Use of Available Treatments for Pediatric Patients With Spinal Muscular Atrophy (SMA): A Multi-Site Retrospective Analysis Based on REaDY Registry

The aim of this study was to evaluate real-world treatment patterns and clinical outcomes in pediatric SMA patients in the Czech and Slovak Republics, using data from the Registry of muscular Dystrophy (REaDY).

Inclusion criteria:

• Patients ≤18 years of age on the index date.
• Patients with genetically confirmed SMA.
• Patients with a known examination on the index date.
• Patients with a signed informed consent from their legal representative.

Exclusion criteria:

None.