Last updated on 18-12-2020 by Marlène Jagut
Scientific poster, presentation or proceeding
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Article written during project(s) :
Samenvatting:
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…