Overview
The BNMDR-SMA project specifically collects data on spinal muscular atrophy patients, on an annual basis. Clinicians from the 7 Belgian neuromuscular reference centers collect robust longitudinal data that (a) captures the natural history of the disease, (b) measures the effectiveness of emerging interventions and (c) informs standards of care for patients.
- Target population: SMA patients living in Belgium, followed at a neuromuscular reference center.
- Data collection started in 2017 and is still ongoing.
RIZIV/INAMI
Demographics
Dataset
The database includes information on selected sociodemographic characteristics (such as age, gender, place of residence and living status), (genetic) diagnosis, family history, clinical observations, scoliosis, motor function, wheelchair use, motor measures, nutrition, pulmonary function, therapies and medications, hospitalisations and comorbidities, participation in clinical research, patient-reported outcomes (PRO), electrophysiology and biomarkers.
Type of data: pseudonymised individual data. Data are not publicly accessible. Specific data (analyses) are provided upon request.
A data request from is in place. Approval of the BNMDR scientific committee is needed. Data requests are discussed with a Group of Experts. An anonymised data set can be shared.
Contact
Rue Juliette Wytsmanstraat 14, 1050 Brussels
https://www.sciensano.be/en