Natural history, outcome measures and trial readiness in LAMA2-related muscular dystrophy and SELENON-related myopathy in children and adults: protocol of the LAST STRONG study

K. Bouman*, J.T. Groothuis, J. Doorduin, N. van Alfen, F.E.A.U. ten Cate, F.M.A. van den Heuvel, R. Nijveldt, W.C.M. van Tilburg, S.C.F.M. Buckens, A.T.M. Dittrich, J.M.T. Draaisma, M.C.H. Janssen, E.J. Kamsteeg, E.S.B. van Kleef, S. Koene, J.A.M. Smeitink, B. Kusters, F.H.J. van Tienen, H.J.M. Smeets, B.G.M. van EngelenC.E. Erasmus, N.C. Voermans

*Corresponding author for this work

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Medicine and Dentistry

Biochemistry, Genetics and Molecular Biology

Neuroscience