SMA CLINICAL DATA, OUTCOME MEASURES AND REGISTRIES

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Spinal muscular atrophy (SMA) is a rare genetic neuromuscular disorder caused by a loss of motor neurons in the spinal cord and the brainstem, leading to muscle weakness and atrophy. The estimated incidence is 1:6,000 to 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=50) collect a common core dataset and are governed by the TREAT-NMD Global Database Oversight Committee (TGDOC).

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