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PLoS ONE, ISSN 1932-6203, 10/2014, Volume 9, Issue 10, p. e108205
Journal Article
Journal Article
PLoS ONE, ISSN 1932-6203, 02/2017, Volume 12, Issue 2, p. e0172346
Journal Article
Neurology, ISSN 0028-3878, 09/2019, Volume 93, Issue 13, pp. e1241 - e1247
OBJECTIVETo report the long-term progression in a cohort of patients with type II spinal muscular atrophy (SMA) assessed with the Hammersmith Functional Motor... 
Journal Article
Neurology, ISSN 0028-3878, 07/2018, Volume 91, Issue 8, pp. e696 - e703
OBJECTIVETo report cross-sectional clinical findings in a large cohort of patients affected by type 1 spinal muscular atrophy. METHODSWe included 122 patients,... 
SHAM CONTROL | NUSINERSEN | THERAPY | SPINAL MUSCULAR-ATROPHY | LONG-TERM SURVIVAL | WERDNIG-HOFFMANN-DISEASE | CLINICAL NEUROLOGY | 176 | 177 | 185
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Neuromuscular Disorders, ISSN 0960-8966, 07/2018, Volume 28, Issue 7, pp. 582 - 585
We report preliminary data on the six month use of Nusinersen in 104 type 1 patients of age ranging from three months to 19 years, 9 months. Ten of the 104... 
Nusinersen | Werdnig Hoffmann disease | Spinal Muscular Atrophy | CHOP INTEND | Hammersmith Infant Neurological Examination | SHAM CONTROL | SPINAL MUSCULAR-ATROPHY | NATURAL-HISTORY | NEUROSCIENCES | CLINICAL NEUROLOGY | AGE | Infants
Journal Article
Journal Article
PLoS ONE, ISSN 1932-6203, 03/2016, Volume 11, Issue 3, p. e0151445
Journal Article