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

Plain-language summary

This is an observational study examining the real-world clinical use of available treatments in pediatric patients with spinal muscular atrophy (SMA). Patients aged 18 and under with a genetically confirmed SMA diagnosis and consent from their legal representative were included. It is a study with no assigned phase and has been completed.

The summary above is a plain-language rendering of the official record. The original English title is shown for reference.

Trial details

Phase

N/A

Status

Completed

Sponsor

Novartis Pharmaceuticals

Start date

11/22/2011

Estimated completion

3/13/2025

Countries

Czechia

Related genes

Locations

• Prague, Czechia

Eligibility criteria

Criteria are reproduced from the ClinicalTrials.gov record in the original English. Only the trial team can determine 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.