Therapeutic Management and Use of Resources and Costs of Spinal Muscular Atrophy in Spain

Novartis

Status: Completed

Conditions: Spinal Muscular Atrophy Type I; Spinal Muscular Atrophy Type II

Study type: Observational

Funder types: Industry

Identifiers

NCT06632730

COAV101A1ES05

Details and patient eligibility

About

An observational, retrospective, cross-sectional, multicenter study. Real-world data were obtained from medical records of Spanish public hospitals (9 hospitals).

Enrollment: 69 patients

Sex: All

Ages: Under 14 years old

Volunteers: No Healthy Volunteers

Inclusion criteria

• Patients diagnosed with SMA type I or SMA type II.
• Patients with medical records available in the hospital.
• Patients diagnosed with SMA type I or SMA type II after 2017 for whom a minimum of 6 months of follow-up was available.

Exclusion criteria

• Patients who participated in an experimental design study except for those in the long-term follow-up period, during the observation period.

Trial design

69 participants in 1 patient group

Spinal Muscular Atrophy (SMA) Cohort

Description:

Pediatric patients with SMA type I, SMA type II, or presymptomatic SMA.