Prospective and longitudinal natural history study of patients with Type 2 and 3 spinal muscular atrophy: Baseline data NatHis-SMA study

Spinal muscular atrophy (SMA) is a monogenic disorder caused by loss of function mutations in the survival motor neuron 1 gene, which results in a broad range of disease severity, from neonatal to adult onset. There is currently a concerted effort to define the natural history of the disease and dev...

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Автори:	Chabanon, A, Seferian, AM, Daron, A, Péréon, Y, Cances, C, Vuillerot, C, De Waele, L, Cuisset, J-M, Laugel, V, Schara, U, Gidaro, T, Gilabert, S, Hogrel, J-Y, Baudin, P-Y, Carlier, P, Fournier, E, Lowes, LP, Hellbach, N, Seabrook, T, Toledano, E, Annoussamy, M, Servais, L
Інші автори:	NatHis-SMA Study Group
Формат:	Journal article
Мова:	English
Опубліковано:	Public Library of Science 2018

Prospective and longitudinal natural history study of patients with Type 2 and 3 spinal muscular atrophy: Baseline data NatHis-SMA study

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