Developmental milestones in type I spinal muscular atrophy

Highlights • The paper reports patterns of natural progression in type 1 SMA. • This is the first time the HINE is used to capture possible changes motor developmental milestones in a neuromuscular population. • Even with current standards of care, motor developmental milestones are rarely acquired,...

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Veröffentlicht in:Neuromuscular disorders : NMD 2016-11, Vol.26 (11), p.754-759
Hauptverfasser: De Sanctis, Roberto, Coratti, Giorgia, Pasternak, Amy, Montes, Jacqueline, Pane, Marika, Mazzone, Elena S, Young, Sally Dunaway, Salazar, Rachel, Quigley, Janet, Pera, Maria C, Antonaci, Laura, Lapenta, Leonardo, Glanzman, Allan M, Tiziano, Danilo, Muntoni, Francesco, Darras, Basil T, De Vivo, Darryl C, Finkel, Richard, Mercuri, Eugenio
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Sprache:eng
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Zusammenfassung:Highlights • The paper reports patterns of natural progression in type 1 SMA. • This is the first time the HINE is used to capture possible changes motor developmental milestones in a neuromuscular population. • Even with current standards of care, motor developmental milestones are rarely acquired, as part of natural history in type I SMA infants.
ISSN:0960-8966
1873-2364
DOI:10.1016/j.nmd.2016.10.002