M.Q., F.P.M.C., S.R. and C.R. conceived and designed the study. M.Q., K.R., K.K. and M.U. analyzed pedigrees and genotypes. J.B., S.H.T., C.L., A.I.J., K.B.F. and W.L. contributed to the collection of material and clinical data for family M1-A. F.P.M.C., S.E.d.B., D.M.P., R.J.H.-M. and S.R. coordinated the collection of DNA samples. K.R., S.E.d.B., E.G.M.B., N.Z., L.K.H., Z. Corradi, S.S., D.M.P. and R.J.H.-M. performed the majority of the Sanger sequencing screening. K.K., A.B.I.-R. and M.F. carried out the molecular biology experiments. M.Q. and C.R. performed the statistical analyses. M.Q. and J.M.E. retrieved and analyzed large-scale data. K.R. and S.R. coordinated the genotyping of the cohorts. W.L. was responsible for clinical data analysis. Z. Cvackova and D. Stanek conducted all in vitro experiments. F.P.M.C., C.R. and S.R. supervised the project. M.Q., K.R., F.P.M.C., J.M.E., D. Stanek, S.R. and C.R. prepared the original draft of the manuscript. M.Q., K.R., S.R. and C.R. reviewed and edited the manuscript. M.A., A.A., S.A., G. Ansari, G. Arno, G.D.N.A., C.A., R.A., S.B., E.B., T.S.B., M.T.S.B., M.B., T.B.-Y., V.B., D.G.B., P.B., F.B.-K., B.B., C.J.F.B., K.B., D.B.-G., A.C.B.-J., K.M.B., C.B.d.R., E.L.C., G.C., F.C., L.C., N.C., P.C.I., L.C.-S., S.P.D., E.D.B., M.D.B., B.d.l.C., J.N.D.R., J.D.Z., R.D., C.-M.D., L.D., J.L.D., G.J.F., N.F., B.J.F., L.F.-C., J.M.F.S., S.G., A.G., J.C.G., C.G., R.G.-D., K.G., S.G.-J., T.B.H., L.H.-W., A.J.H., T.H., E.H., L.H.H., A.H., J.P.H., C.B.H., M.B.B.I., C.F.I., T.I., B.O.J., K.J., V.K., S. Kamakari, M.K., U.K., C.C.W.K., K.K., R.K.K., S. Kohl, T.K., L.K., T.M.L., R.L., B.P.L., S.L., P.L., I.L., V.R.d.J.L.-R., Q.M., O.A.M., G.M., L. Mansard, M.P.M.-G., N.M., L. Mauring, M. McKibbin, T.L.M., I.M., M. Michaelides, J.M.M., K.M., R.M., Z.Z.N., K.N., M. Ołdak, M. Oorsprong, Y.P., A. Papachristou, A. Percesepe, M.P., E.A.P., E.P., R.R., F.R., F.A.R., G.I.R., L.R., M.R.-H., J.R.-E., A.H.S., A.F.S., A.I.S.-B., A.S.S., R.S., C.M.S., M. Scarpato, H.P.N.S., D. Sharon, S.G.S., F.S., A.B.S., M. Stefaniotou, K. Stefansson, K. Stingl, A.S., P.S., L.S.S., V.S., J.P.S., G.T., A.A.H.J.T., C.T., V.H.T., M.K.T., P.T., V.V., M.V., S. Valeina, E.M.V., C.V., R.V., S. Valleix, J.v.A., L.I.v.d.B., M.V.H., V.J.M.V., A.L.V., A.R.W., L.W., B.W., G.G.Y., K.Y., J.C.Z., R.Z. and T.Z. were instrumental in acquiring funding, clinical data, validation of genetic or molecular data, and reviewing and editing the final manuscript. All authors approved the final content of this work.