Cerebellar ataxia and congenital disorder of glycosylation Ia (CDG-Ia) with normal routine CDG screening
Cerebellar ataxia and congenital disorder of glycosylation Ia (CDG-Ia) with normal routine CDG screening
Cerebellar ataxia can have many genetic causes among which are the congenital disorders of glycosylation type I (CDG-I). In this group of disorders, a multisystem phenotype is generally observed including the involvement of many organs, the endocrine, hematologic and central nervous systems. A few cases of CDG-Ia have been reported with a milder presentation, namely cerebellar hypoplasia as an isolated abnormality. To identify patients with a glycosylation disorder, isofocusing of plasma transferrin is routinely performed. Here, we describe two CDG-Ia patients,who presented with mainly ataxia and cerebellar hypoplasia and with a normal or only slightly abnormal transferrin isofocusing result. Surprisingly, the activity of the corresponding enzyme phosphomannomutase was clearly deficient in both leucocytes and fibroblasts. Therefore, in patients presenting with apparently recessive inherited ataxia caused by cerebellar hypoplasia and an unknown genetic aetiology after proper diagnostic work-up, we recommend the measurement of phosphomannomutase activity when transferrin isofocusing is normal or inconclusive.
- Radboud University Nijmegen Netherlands
- Radboud University Nijmegen Medical Centre Netherlands
- Rijnstate Hospital Netherlands
- KU Leuven Belgium
Male, DCN 1: Perception and Action, Adolescent, Cerebellar Ataxia, DCN 2: Functional Neurogenomics, NCMLS 5: Membrane transport and intracellular motility, UMCN 5.1: Genetic defects of metabolism, IGMD 3: Genomic disorders and inherited multi-system disorders, Congenital Disorders of Glycosylation, DCN 3: Neuroinformatics, Humans, Child, UMCN 3.2: Cognitive neurosciences, IGMD 4: Glycostation disorders, IGMD 9: Renal disorder, Transferrin, Fibroblasts, Phosphotransferases (Phosphomutases), NCMLS 4: Energy and redox metabolism, Female, IGMD 8: Mitochondrial medicine, Isoelectric Focusing, IGMD 5: Health aging / healthy living
Male, DCN 1: Perception and Action, Adolescent, Cerebellar Ataxia, DCN 2: Functional Neurogenomics, NCMLS 5: Membrane transport and intracellular motility, UMCN 5.1: Genetic defects of metabolism, IGMD 3: Genomic disorders and inherited multi-system disorders, Congenital Disorders of Glycosylation, DCN 3: Neuroinformatics, Humans, Child, UMCN 3.2: Cognitive neurosciences, IGMD 4: Glycostation disorders, IGMD 9: Renal disorder, Transferrin, Fibroblasts, Phosphotransferases (Phosphomutases), NCMLS 4: Energy and redox metabolism, Female, IGMD 8: Mitochondrial medicine, Isoelectric Focusing, IGMD 5: Health aging / healthy living
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