(A) Images of the thigh and calf muscles of the patients. P1 (at 28 years) exhibited atrophy and mild fatty infiltration in thigh muscles, but the triceps surae muscles were rather normal. P2 (at 19 years) and P3 (at 14 years) had sufficient skeletal muscle volumes and no fatty change. P1 and P3: T1-weighted image; P2: CT image. (B) Histology and immunostaining of skeletal muscle from the patients. The muscle histology in P1 exhibited a muscular dystrophy–like appearance, including necrotic and regenerating fibers, fibers with internal nuclei, and mild endomysial fibrosis, whereas those of P3 were mild and of P2 were almost normal except for some fibers with internal nuclei according to hematoxylin and eosin staining (HE). All muscles were positive for antibodies to β-dystroglycan (β-DG), but negative for the glycoepitope antibody to α-dystroglycan (α-DG VIA4-1). (C) Western blotting with VIA4-1 antibody and the laminin overlay assay of muscle proteins detected 2 reactive bands of α-DG at 150 and 120 kDa, whereas the GT20ADG antibody against the core peptide detected an additional band at 100 kDa. β-DG staining was normal.