Patterns of disease progression in type 2 and 3 SMA: Implications for clinical trials
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- 作者：Eugenio Mercuria ; 1 ; mercuri@rm.unicatt.it" class="auth_mail" title="E-mail the corresponding author ; Richard Finkelb ; 1 ; Jacqueline Montesc ; Elena S. Mazzonea ; Maria Pia Sormanid ; Marion Maine ; Danielle Ramseye ; Anna Mayhewf ; Allan M. Glanzmang ; Sally Dunawayc ; Rachel Salazarc ; Amy Pasternakh ; Janet Quigleyh ; Marika Panea ; Maria Carmela Peraa ; Mariacristina Scotoe ; Sonia Messinai ; Maria Sframelii ; Gian Luca Vitai ; Adele D'Amicoj ; Marleen van den Hauwek ; Serena Sivoa ; Nathalie Goemansk ; Petra Kaufmannc ; Basil T. Darrash ; Enrico Bertinij ; Francesco Muntonie ; 2 ; Darryl C. De Vivoc ; 2
- 关键词：Spinal muscular atrophy ; Hammersmith Functional Motor Scale ; Outcome measures
- 刊名：Neuromuscular Disorders
- 出版年：2016
- 出版时间：February 2016
- 年：2016
- 卷：26
- 期：2
- 页码：126-131
- 全文大小：824 K
文摘
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The paper reports for the first time patterns of progression in type 2 and 3 SMA.
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Different trajectories can be identified in ambulant and non-ambulant patients.
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Age appears to be an important factor in determining trajectories of progression.