General SMA project information

Last updated: 2023-03-21 10:48

Project name

Belgian Neuromuscular Diseases Registry: Spinal Muscular Atrophy

Project abbreviation

BNMDR-SMA

Project code

HDBP0198

Primary organization that oversees implementation of project

  • Sciensano

Partner organization participating in project

  • Not available

Organization that commissioned this project

  • National Institute for Health and Disability Insurance (RIZIV-INAMI)

Organization providing monetary or material support

  • National Institute for Health and Disability Insurance (RIZIV-INAMI)

Brief project description

Spinal muscular atrophy (SMA) is a rare genetic neuromuscular disorder characterized by a loss of motor neurons in the spinal cord and the brainstem, leading to muscle weakness and atrophy. The estimated incidence is around 1:10,000 live births.

TREAT-NMD is a neuromuscular network that aims to ensure that the most promising new therapies reach patients as quickly as possible. The TREAT-NMD Global Network of SMA Registries (n=49) collects a common core dataset and is governed by the TREAT-NMD Global Database Oversight Committee (TGDOC). Researchers and industry can request anonymised and aggregated data via the committee, offering a single point of access to this diverse and extensive dataset.

The TREAT-NMD SMA core dataset containing 23 data items was established in 2008 when the main purpose of the registries was clinical trial readiness and recruitment. In the current SMA landscape there is a need for more widespread longitudinal data collection to support future research and post marketing surveillance (PMS) requirements for emerging therapies. With this in mind TREAT-NMD reviewed and expanded the core dataset for their SMA Registries.

Regulatory framework of this project

Consult the regulatory framework information published on the fair.healthdata.be pages.