Longitudinal changes in clinical outcome measures in COL6-related dystrophies and LAMA2-related dystrophies

Minal S Jain¹, Katherine Meilleur², Eunhee Kim²+42

• ¹From the Rehabilitation Medicine Department (M.S.J., M.W., R.V., R.L., C.N.), Clinical Research Center, Neuromuscular Symptoms Unit (K.M., I.C., M.L., M.P., J.E., F.T., J.W.), Tissue Injury Branch, National Institute of Nursing Research, Clinical Trials Unit (E.K., G.N.) and Neuromuscular and Neurogenetic Disorders of Childhood Section (S.D., M.L., E.H., G.M.A., A.K., A.S., K.Z., P.M., D.B.-G., A.R.F., C.G.B.), Neurogenetics Branch, National Institute of Neurological Disorders and Stroke, and Pulmonary Branch (M.B., J.F., P.M.), National Heart, Lung, and Blood Institute, NIH, Bethesda, MD; Departments of Physical Therapy (L.N.) and Clinical Sciences (L.H.), University of Texas Southwestern, Dallas; Occupational Therapy and Physical Therapy Department (M. McGuire, J.C.C.), Cincinnati Children's Hospital Medical Center, OH; Pediatric Rehabilitation Medicine Department (T.D.), Children's National Medical Center, Washington, DC; Physical Therapy Department (K.K.), Rady's Children Hospital, San Diego, CA; Department of Physical Therapy (D.J.L.), University of Florida, Gainesville; Physical Therapy Department (A.G.), Children's Hospital of Philadelphia, PA; Paediatric Gait Analysis Laboratory of New South Wales (K.R.), The Children's Hospital at Westmead, Sydney, Australia; Dubowitz Neuromuscular Centre (M. Main), Great Ormond Street Hospital for Children, London, UK; Department of Physical Therapy (C.F.), Kennedy Krieger Institute, Baltimore, MD; G.H. Sergievsky Center and Department of Neurology (R.F., V.H.), Columbia University, New York, NY; Goryeb Children's Hospital (J.D.), Morristown, NJ; CureCMD (E.S.K., A.R.), Torrance, CA; and L'Escale Service Central de MPR pédiatrique (C.V.), Hospices Civils de Lyon, France carsten.bonnemann@nih.gov mjain@cc.nih.gov.+1