The RESTORE Registry: Real -World Assessments of Interventions and Long -Term Outcomes in Patients with Spinal Muscular Atrophy (SMA)

被引：0
|
作者：
Finkel, Richard [1 ]
Day, John [2 ]
De Vivo, Darryl [3 ]
Mercuri, Eugenio [4 ,5 ]
Muntoni, Francesco [6 ,7 ]
Shieh, Perry [8 ]
Tizzano, Eduardo [9 ]
Desguerre, Isabelle [10 ]
Saito, Kayoko [11 ]
Menier, Melissa [12 ]
LaMarca, Nicole [13 ]
Anderson, Fred [14 ]
Dabbous, Omar [13 ]
Servais, Laurent [15 ]
机构：
[1] St Jude Childrens Res Hosp, Ctr Expt Neurotherapeut, 332 N Lauderdale St, Memphis, TN 38105 USA
[2] Stanford Univ, Sch Med, Dept Neurol, Stanford, CA 94305 USA
[3] Columbia Univ Irving Med Ctr, Neurol & Pediat, New York, NY USA
[4] Catholic Univ, Dept Paediat Neurol, Rome, Italy
[5] Catholic Univ, Nemo Clin Ctr, Rome, Italy
[6] UCL Inst Child Hlth, London, England
[7] Great Ormond St Hosp Biomed Res Ctr, Natl Inst Hlth Res, London, England
[8] Univ Calif Los Angeles, David Geffen Sch Med, Dept Neurol, Los Angeles, CA 90024 USA
[9] Hosp Valle De Hebron, Dept Clin & Mol Genet, Barcelona, Spain
[10] Hop Necker Enfants Malad, AP HP, Paris, France
[11] Tokyo Womens Med Univ, Inst Med Genet, Tokyo, Japan
[12] Atlas Clar LLC, Perth Amboy, NJ USA
[13] Novartis Gene Therapies Inc, Bannockburn, IL USA
[14] Univ Massachusetts, Med Sch, Ctr Outcomes Res, Amherst, MA 01003 USA
[15] Univ Oxford, MDUK Oxford Neuromuscular Ctr, Oxford, England
来源：
NEUROLOGY | 2022年 / 98卷 / 18期
关键词：
D O I：
暂无
中图分类号：
R74 [神经病学与精神病学];
学科分类号：
摘要：
P15-5.002
引用
收藏
页数：5
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