Spinal Muscular Atrophies
Conditions
Interventions
This is a study observational, longitudinal, ambispective cohort designed to collect real-world data on the effectiveness, safety and costs associated with Onasemnogene Abeparvovec (Zolgensma). Follow
E05.318.372.250.500
Sponsors
Hospital Universitário Professor Edgard Santos da Universidade Federal da Bahia
Hospital Universitário Professor Edgard Santos da Universidade Federal da Bahia
Eligibility
Age
No minimum to 10 Years
Inclusion criteria
Inclusion criteria: Confirmed genetic diagnosis of Atrofia Muscular Espinhal 5q type 1 or type 2; have previously received or have a confirmed court order for the use of the onasemnogene abeparvovec-xioi (Zolgensma), even if older than 24 months
Exclusion criteria
Exclusion criteria: Inability to locate children based on the data provided by the Ministry of Health. No other exclusion criteria are anticipated
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| We aim to identify changes in the scores of the scales and instruments recommended by the National Commission for the Incorporation of Technologies into the Unified Health System, obtain data on the utilization and cost of financial resources, measure neurodevelopmental outcomes related to nutrition and anthropometric status, patient-reported outcomes, and safety outcomes, such as the occurrence of adverse events secondary to gene therapy | — |
Secondary
| Measure | Time frame |
|---|---|
| Evaluate the use of other therapies for Spinal Muscular Atrophy and the conditions and quality of access to health resources | — |
Countries
Brazil
Contacts
Public ContactNey Boa Sorte
Hospital Universitário Professor Edgard Santos da Universidade Federal da Bahia
Outcome results
None listed