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Siblings with Glutaric Aciduria Type 1 with Atypical Phenotype with Novel Pathogenic Variant in GCDH Gene

Glutaric aciduria type 1 (GA1) is caused by a deficiency of the enzyme glutaryl CoA dehydrogenase. It generally presents with developmental delay, dystonia, and large head. We are reporting siblings of GA1, presenting with an atypical phenotype with novel pathogenic variant. Thirteen-year-old boy pr...

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Dades bibliogràfiques
Publicat a:J Pediatr Neurosci
Autors principals: Gowda, Vykuntaraju Kammasandra, Nagarajan, Balamurugan, Srinivasan, Varunvenkat M., Bhat, Maya
Format: Artigo
Idioma:Inglês
Publicat: Wolters Kluwer - Medknow 2021
Matèries:
Accés en línia:https://ncbi.nlm.nih.gov/pmc/articles/PMC8276965/
https://ncbi.nlm.nih.gov/pubmed/34316315
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.4103/jpn.JPN_63_20
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