Niemann–Pick disease type C (a cellular cholesterol lipidosis) treated by bone marrow transplantation

  title={Niemann–Pick disease type C (a cellular cholesterol lipidosis) treated by bone marrow transplantation},
  author={Y M Hsu and W. L. Hwu and S Huang and M Lu and R-L Chen and D Lin and SSF Peng and Kwang-Huei Lin},
  journal={Bone Marrow Transplantation},
Bone marrow transplantation (BMT) has been used for a wide variety of lysosomal storage diseases with encouraging results. We report a 3-year 5-month-old girl with Niemann–Pick type C disease (NPC) who received an allogeneic BMT. The patient presented with repeated lower respiratory tract infections, hepatosplenomegaly, failure to thrive, and developmental delay. Chest computed tomography (CT) revealed diffuse interstitial lung infiltration. Bone marrow and liver biopsies revealed abundant… CONTINUE READING
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