Two novel variants in CNTNAP1 in two siblings presenting with congenital hypotonia and hypomyelinating neuropathy
Two novel variants in CNTNAP1 in two siblings presenting with congenital hypotonia and hypomyelinating neuropathy
Homozygous frameshift variants in CNTNAP1 have recently been reported in patients with arthrogryposis and abnormal axon myelination. In two brothers with severe congenital hypotonia and foot deformities, we identified compound heterozygous variants in CNTNAP1, reporting the first causative missense variant, p.(Cys323Arg). Motor nerve conductions were markedly decreased. Nerve microscopical lesions confirmed a severe hypomyelinating process and showed loss of attachment sites of the myelin loops on the axons, which could be a characteristic of Caspr loss-of-function. We discuss the pathophysiology of the myelination process and we propose to consider this disorder as a congenital hypomyelinating neuropathy.
Arthrogryposis, Foot Deformities, Male, [SDV.MHEP.PED]Life Sciences [q-bio]/Human health and pathology/Pediatrics, neuropathies, Cell Adhesion Molecules, Neuronal, Siblings, Homozygote, Infant, Newborn, Mutation, Missense, 610, Infant, [SDV.MHEP.PED] Life Sciences [q-bio]/Human health and pathology/Pediatrics, factor, Humans, Muscle Hypotonia, Genetic Predisposition to Disease, genetic, Myelin Sheath, congenita
Arthrogryposis, Foot Deformities, Male, [SDV.MHEP.PED]Life Sciences [q-bio]/Human health and pathology/Pediatrics, neuropathies, Cell Adhesion Molecules, Neuronal, Siblings, Homozygote, Infant, Newborn, Mutation, Missense, 610, Infant, [SDV.MHEP.PED] Life Sciences [q-bio]/Human health and pathology/Pediatrics, factor, Humans, Muscle Hypotonia, Genetic Predisposition to Disease, genetic, Myelin Sheath, congenita
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