<?xml version="1.0" encoding="UTF-8"?><xml><records><record><source-app name="Biblio" version="7.x">Drupal-Biblio</source-app><ref-type>13</ref-type><contributors><authors><author><style face="normal" font="default" size="100%">Miriam Rodrigues</style></author><author><style face="normal" font="default" size="100%">JoAnne Bullivant</style></author><author><style face="normal" font="default" size="100%">Victoria Hodgkinson</style></author><author><style face="normal" font="default" size="100%">TREAT-NMD Registry Curators</style></author><author><style face="normal" font="default" size="100%">Marlène Jagut</style></author><author><style face="normal" font="default" size="100%">Volker Straub</style></author><author><style face="normal" font="default" size="100%">Nathalie Goemans</style></author><author><style face="normal" font="default" size="100%">Anna Ambrosini</style></author><author><style face="normal" font="default" size="100%">Craig Campbell</style></author></authors></contributors><titles><title><style face="normal" font="default" size="100%">Collaborative data collection by TREAT-NMD Registries to support post-marketing surveillance in Spinal Muscular Atrophy</style></title><secondary-title><style face="normal" font="default" size="100%">TREAT-NMD conference</style></secondary-title></titles><keywords><keyword><style  face="normal" font="default" size="100%">Data collection</style></keyword><keyword><style  face="normal" font="default" size="100%">post-marketing surveillance</style></keyword><keyword><style  face="normal" font="default" size="100%">SMA</style></keyword><keyword><style  face="normal" font="default" size="100%">TREAT-NMD</style></keyword></keywords><dates><year><style  face="normal" font="default" size="100%">2019</style></year><pub-dates><date><style  face="normal" font="default" size="100%">12/2019</style></date></pub-dates></dates><publisher><style face="normal" font="default" size="100%">TREAT-NMD</style></publisher><pub-location><style face="normal" font="default" size="100%">Leiden, The Netherlands</style></pub-location><language><style face="normal" font="default" size="100%">eng</style></language><abstract><style face="normal" font="default" size="100%">&lt;p&gt;Spinal muscular atrophy (SMA) is a rare genetic neuromuscular disorder&lt;br&gt;
characterized by a loss of motor neurons in the spinal cord and the brainstem,&lt;br&gt;
leading to muscle weakness and atrophy. The estimated incidence is around&lt;br&gt;
1:10,000 live births.&lt;br&gt;
TREAT-NMD is a neuromuscular network that aims to ensure that the most&lt;br&gt;
promising new therapies reach patients as quickly as possible.&lt;br&gt;
The TREAT-NMD Global Network of SMA Registries (n=49) collects a common&lt;br&gt;
core dataset and is governed by the TREAT-NMD Global Database Oversight&lt;br&gt;
Committee (TGDOC). Researchers and industry can request anonymised and&lt;br&gt;
aggregated data via the committee, offering a single point of access to this&lt;br&gt;
diverse and extensive dataset.&lt;br&gt;
The TREAT-NMD SMA core dataset containing 23 data items was established&lt;br&gt;
in 2008 when the main purpose of the registries was clinical trial readiness&lt;br&gt;
and recruitment. In the current SMA landscape there is a need for more&lt;br&gt;
widespread longitudinal data collection to support future research and post&lt;br&gt;
marketing surveillance (PMS) requirements for emerging therapies. With this&lt;br&gt;
in mind TREAT-NMD reviewed and expanded the core dataset for their SMA&lt;br&gt;
Registries.&lt;br&gt;
&amp;nbsp;&lt;/p&gt;
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