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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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Dettagli Bibliografici
Pubblicato in:J Pediatr Neurosci
Autori principali: Gowda, Vykuntaraju Kammasandra, Nagarajan, Balamurugan, Srinivasan, Varunvenkat M., Bhat, Maya
Natura: Artigo
Lingua:Inglês
Pubblicazione: Wolters Kluwer - Medknow 2021
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Accesso online: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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