Patterns of disease progression in type 2 and 3 SMA: Implications for clinical trials
Highlights • The paper reports for the first time patterns of progression in type 2 and 3 SMA. • Different trajectories can be identified in ambulant and non-ambulant patients. • Age appears to be an important factor in determining trajectories of progression.
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| Veröffentlicht in: | Neuromuscular disorders : NMD 2016-02, Vol.26 (2), p.126-131 |
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| container_title | Neuromuscular disorders : NMD |
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| creator | Mercuri, Eugenio Finkel, Richard Montes, Jacqueline Mazzone, Elena S Sormani, Maria Pia Main, Marion Ramsey, Danielle Mayhew, Anna Glanzman, Allan M Dunaway, Sally Salazar, Rachel Pasternak, Amy Quigley, Janet Pane, Marika Pera, Maria Carmela Scoto, Mariacristina Messina, Sonia Sframeli, Maria Vita, Gian Luca D'Amico, Adele van den Hauwe, Marleen Sivo, Serena Goemans, Nathalie Kaufmann, Petra Darras, Basil T Bertini, Enrico Muntoni, Francesco De Vivo, Darryl C |
| description | Highlights • The paper reports for the first time patterns of progression in type 2 and 3 SMA. • Different trajectories can be identified in ambulant and non-ambulant patients. • Age appears to be an important factor in determining trajectories of progression. |
| doi_str_mv | 10.1016/j.nmd.2015.10.006 |
| format | Article |
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| identifier | ISSN: 0960-8966 |
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| source | MEDLINE; ScienceDirect Journals (5 years ago - present) |
| subjects | Adolescent Adult Child Child, Preschool Clinical Trials as Topic Disease Progression Female Hammersmith Functional Motor Scale Humans Male Middle Aged Mobility Limitation Neurology Outcome measures Retrospective Studies Spinal Muscular Atrophies of Childhood - physiopathology Spinal muscular atrophy Young Adult |
| title | Patterns of disease progression in type 2 and 3 SMA: Implications for clinical trials |
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