Publications
HaematologicaNov 2025 |
110
(
11
),
2819-2822
DOI:
10.3324/haematol.2025.287393

Myopathy, lactic acidosis and sideroblastic anemia syndrome 1 (MLASA1): clinical hallmarks in a large pedigree with a novel PUS1 R144Q mutation, remarkable response to somatropin, and review of the literature

Parisi, Lorenzo; Escher, Robert
Product Used
NGS
Product Used
NGS

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