Novel Translational Read-through–Inducing Drugs as a Therapeutic Option for Shwachman-Diamond Syndrome
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DOI: 10.3390/biomedicines10040886
Abstract: Shwachman-Diamond syndrome (SDS) is one of the most commonly inherited bone marrow failure syndromes (IBMFS). In SDS, bone marrow is hypocellular, with marked neutropenia. Moreover, SDS patients have a high risk of developing myelodysplastic syndrome… read more here.
Keywords: diamond syndrome; translational read; bone marrow; sds ... See more keywords