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Drug Design and Development for Rare Hematologic Diseases

Bruno Fattizzo1,2, Marco Capecchi3,4, Irene Motta5,6

  • 1SC Ematologia, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, 20122 Milan, Italy.

Pharmaceuticals (Basel, Switzerland)
|October 28, 2023
PubMed

Abstract:

The last decade has seen an exponential increase in therapeutic options for rare hematologic diseases [...].

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Bone marrow transplant is a potential cure for several diseases, including cancer and specific genetic disorders. Notably, this procedure is applicable for patients suffering from aplastic anemia, certain types of leukemia, severe combined immunodeficiency disease (SCID), Hodgkin's disease, non-Hodgkin's lymphoma, multiple myeloma, thalassemia, sickle-cell disease, and certain cancers.
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