Ontology highlight
ABSTRACT:
SUBMITTER: Kolb SJ
PROVIDER: S-EPMC5776712 | biostudies-literature | 2017 Dec
REPOSITORIES: biostudies-literature
Kolb Stephen J SJ Coffey Christopher S CS Yankey Jon W JW Krosschell Kristin K Arnold W David WD Rutkove Seward B SB Swoboda Kathryn J KJ Reyna Sandra P SP Sakonju Ai A Darras Basil T BT Shell Richard R Kuntz Nancy N Castro Diana D Parsons Julie J Connolly Anne M AM Chiriboga Claudia A CA McDonald Craig C Burnette W Bryan WB Werner Klaus K Thangarajh Mathula M Shieh Perry B PB Finanger Erika E Cudkowicz Merit E ME McGovern Michelle M MM McNeil D Elizabeth DE Finkel Richard R Iannaccone Susan T ST Kaye Edward E Kingsley Allison A Renusch Samantha R SR McGovern Vicki L VL Wang Xueqian X Zaworski Phillip G PG Prior Thomas W TW Burghes Arthur H M AHM Bartlett Amy A Kissel John T JT
Annals of neurology 20171208 6
<h4>Objective</h4>Infantile-onset spinal muscular atrophy (SMA) is the most common genetic cause of infant mortality, typically resulting in death preceding age 2. Clinical trials in this population require an understanding of disease progression and identification of meaningful biomarkers to hasten therapeutic development and predict outcomes.<h4>Methods</h4>A longitudinal, multicenter, prospective natural history study enrolled 26 SMA infants and 27 control infants aged <6 months. Recruitment ...[more]