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

About This Study

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).

Conditions Studied

Eligibility

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.

Study Locations (1)