In conclusion, despite the fact that the precise molecular mechanism/s linking SBDS protein to ERK1/2 activation remain obscure and will represent a future subject of investigation, our findings open a wider therapeutic scenario within SDS pathology, providing a new rationale to promote the evaluation of drugs targeting mTOR pathway in SDS patients with the aim to reduce the risk to progression to bone marrow failure, myelodysplasia and leukemic transformation thus avoiding or postponing the need of bone marrow transplantation. This evidence concerns the gene MAPK3 and Bone marrow hypocellularity.