Article
Results of a two-year pilot study of clinical outcome measures in collagen VI- and laminin alpha2-related congenital muscular dystrophies.
Neuromuscular disorders : NMD - 1 Jan 2015
Meilleur Katherine G, Jain Minal S, Hynan Linda S, Shieh Ching-Yi, Kim Eunice, Waite Melissa, McGuire Michelle, Fiorini Courtney, Glanzman Allan M, Main Marion, Rose Kristy, Duong Tina, Bendixen Roxanna, Linton Melody M, Arveson Irene C, Nichols Carmel, Yang Kelly, Fischbeck Kenneth H, Wagner Kathryn R, North Kathryn, Mankodi Ami, Grunseich Christopher, Hartnett Elizabeth J, Smith Michaele, Donkervoort Sandra, Schindler Alice, Kokkinis Angela, Leach Meganne, Foley A Reghan, Collins James, Muntoni Francesco, Rutkowski Anne, Bönnemann Carsten G
Abstract excerpt
Potential therapies are currently under development for two congenital muscular dystrophy (CMD) subtypes: collagen VI-related muscular dystrophy (COL6-RD) and laminin alpha 2-related dystrophy (LAMA2-RD). However, appropriate clinical outcome measures to be used in clinical trials have not been validated in CMDs. We conducted a two-year pilot study to evaluate feasibility, reliability, and validity of various...
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