Collaborative data collection by TREAT-NMD Registries to support post-marketing surveillance in Spinal Muscular Atrophy

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.