A novel variant in ARSA causes a rare phenotype of infantile metachromatic leukodystrophy in a Malian family.

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Tác giả: Mohamed Albakaye, Abdoulaye Arama, Salia Bamba, Salimata Diarra, Samba Ogomaly Djimdé, Modibo K Goita, Mustafa K Khokha, Mahamadou Kotioumbé, Saquib A Lakhani, Guida Landouré, Alassane Baneye Maiga, Moussa Sangaré, Oumou Traoré, Ji Weizhen, Abdoulaye Yalcouyé

Ngôn ngữ: eng

Ký hiệu phân loại: 230.071 Education in Christianity, in Christian theology

Thông tin xuất bản: United States : Neurogenetics , 2025

Mô tả vật lý:

Bộ sưu tập: NCBI

ID: 694526

 Metachromatic Leukodystrophy (MLD) is a rare autosomal recessive lysosomal disorder caused by ARSA gene variants, affecting central and peripheral nervous systems. While ARSA variants are reported globally, data from sub-Saharan Africa (SSA) are limited. We report the first SSA case, a Malian patient with a rare phenotype: predominant tonic seizures without typical peripheral neuropathy signs. The patient harbored a novel ARSA variant (c.191T >
  C
  p.Phe64Ser), predicted deleterious by in silico tools. This case expands the genetic and phenotypic spectrum of MLD, underscoring the need for genetic studies in underrepresented regions.
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