Results of a two-year pilot study of clinical outcome measures in collagen VI- and laminin alpha2-related congenital muscular dystrophies
Katherine G Meilleur, Minal S Jain, Linda S Hynan, Ching-Yi Shieh, Eunice Kim, Melissa Waite, Michelle McGuire, Courtney Fiorini, Allan M Glanzman, Marion Main, Kristy Rose, Tina Duong, Roxanna Bendixen, Melody M Linton, Irene C Arveson, Carmel Nichols, Kelly Yang, Kenneth H Fischbeck, Kathryn R Wagner, Kathryn North Show all
Neuromuscular Disorders | PERGAMON-ELSEVIER SCIENCE LTD | Published : 2015
Awarded by Medical Research Council
Awarded by Muscular Dystrophy UK
Awarded by NATIONAL INSTITUTE OF NEUROLOGICAL DISORDERS AND STROKE
Awarded by NATIONAL INSTITUTE OF NURSING RESEARCH
We would like to thank the following people without which this study would not have been possible: the participants and their families for their time, flexibility, and commitment during the two years of this study; the nurses and staff of the NIH Clinical Center's outpatient neurology clinic (year 1) and pediatric outpatient clinic (year 2); the National Heart Lung and Blood Institute's Pulmonary Function Lab physicians, therapists, and staff; Mrs. Livija Medne for her assistance with the study in year 2; Dr. Carole Vuillerot and Dr. Joan Austin for their critical feedback on the manuscript. This work was supported by Cure CMD and the NIH intramural funds of the NINDS, NINR, and Mark O. Hatfield Clinical Research Center.