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

CompletedN/A

Sponsor: Novartis Pharmaceuticals

Conditions: Muscular Atrophy, Spinal

Countries: Czechia

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).

Eligibility overview

Sex: ALL

Age: — to 18 Years

Healthy volunteers: No

Study type: OBSERVATIONAL

Eligibility criteria

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.

Locations (1)

Prague, Czechia