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

Last updated on 18-12-2020 by Marlène Jagut

Scientific poster, presentation or proceeding



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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 anonymise…

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