Abstract: The present invention relates to the use of hydroxycarbamide (HC) for reducing and/or delaying the extension of capillary nonperfusion, a cause of irreparable visual impairment in patients suffering from central retinal vein occlusion (CRVO). This is the first systemic treatment which makes it possible to reduce retinal ischemic complications in patients in whom (CRVO) has been recently diagnosed and is consequently in a rapidly progressive phase. Given the low toxicity of HC evaluated on a large scale in children and adults in the context of other diseases for decades, the results of the present study open up a new therapeutic approach in the treatment of CRVO.

Abstract: The present invention relates to antagonists of GDF11, for use in the treatment of malignant hematological disease, such as Acute Myeloid Leukemia (AML). The present invention also relates to a method for predicting the responsiveness of a patient affected with malignant haematological disease, such as Acute Myeloid Leukemia, to a chemotherapy treatment.

Abstract: The present invention relates to methods and pharmaceutical compositions for the treatment of beta-thalassemias. In particular, the present invention relates to an XPO1 inhibitor for use in a method for treating beta-thalassemia in a subject in need thereof.

Abstract: The present invention provides methods and pharmaceutical compositions designed to intervene in this defective process and to promote or restore erythrocyte maturation in individuals suffering from a myelodysplastic syndrome. The methods involve maintaining the activity of GATA-1 by preventing sequestration of Hsp70 in the cytoplasm. Accordingly, it is an object of this invention to provide methods of restoring or increasing erythrocyte maturation in a subject suffering from a myelodysplastic syndrome by preventing proteolytic inactivation of GATA-1. In some embodiments, preventing is achieved by administering to the subject a compound that inhibits the XPO1 nuclear transporter.

Abstract: The present invention concerns a combination of (i) a DNA methylation inhibitor, and (ii) a Vitamin D receptor agonist, for simultaneous or sequential use in the treatment of a drug resistant cancer and/or in prevention of tumor relapse in a patient suffering from cancer. The present invention also relates to a combination of (i) a DNA methylation inhibitor, and (ii) a Vitamin D receptor agonist, for increasing, restoring or enhancing sensitivity of a patient suffering from cancer to a chemotherapeutic drug in a patient suffering from cancer.

Abstract: The present invention provides methods and pharmaceutical compositions designed to intervene in this defective process and to promote or restore erythrocyte maturation in individuals suffering from a myelodysplastic syndrome. The methods involve maintaining the activity of GATA-1 by preventing sequestration of Hsp70 in the cytoplasm. Accordingly, it is an object of this invention to provide methods of restoring or increasing erythrocyte maturation in a subject suffering from a myelodysplastic syndrome by preventing proteolytic inactivation of GATA-1. In some embodiments, preventing is achieved by administering to the subject a compound that inhibits the XPO1 nuclear transporter.

Abstract: The present invention relates to methods and pharmaceutical compositions for the treatment of diseases mediated by the NRP-1/OBR complex signaling pathway. In particular, the present invention relates to a method for treating a disease selected from the group consisting of cancers, obesity and obesity related diseases, anorexia, autoimmune diseases and infectious diseases in a subject in need thereof comprising administering the subject with a therapeutically effective amount of an antagonist of the NRP-1/OBR signaling pathway.

Abstract: The present invention relates to methods and pharmaceutical compositions for the treatment of beta-thalassemias. In particular, the present invention relates to an XPO1 inhibitor for use in a method for treating beta-thalassemia in a subject in need thereof.

Abstract: Methods and compositions for the treatment of ?-thalassemia are provided. Methods and compositions restore or increase erythrocyte maturation in individuals afflicted with ?-TM by preventing proteolysis of GATA-1 protein. Screening methods for identifying agents which bind heat shock protein 70 (HSP-70) and inhibit HSP-70 ?-globin binding, but which allow GATA-1 protein-HSP-1 binding in a manner that prevents GATA-1 proteolysis.

Abstract: The present invention relates to methods and compositions for the treatment of retinal vein occlusion.

Abstract: Methods and compositions for the treatment of ?-thalassemia are provided. Methods and compositions restore or increase erythrocyte maturation in individuals afflicted with ?-TM by preventing proteolysis of GATA-1 protein. Screening methods for identifying agents which bind heat shock protein 70 (HSP-70) and inhibit HSP-70 ?-globin binding, but which allow GATA-1 protein-HSP-1 binding in a manner that prevents GATA-1 proteolysis.

Abstract: The present invention relates to a method for determining whether a candidate human transplant donor is at risk of inducing acute graft versus host disease (aGVHD) in a human transplant recipient, which may in turn allow the selection of a donor exhibiting no risk for the recipient. The present invention also relates to a method for adjusting the immunosuppressive treatment administered to a human transplanted recipient following its graft transplantation after having performing the method for determining risk of the invention. The methods comprise expanding the candidate donor's iNKT cells (invariant NKT cells) and determining the presence or absence of expansion of the CD4(?) iNKT cell sub-population. In particular, CD3+CD4? TCRV[alpha]24V[beta]11 cells are determined. Kits are disclosed.

Abstract: Provided herein are methods of treating diseases associated with anemia or ineffective erythropoiesis by using the level and/or activity of a ligand of an activin receptor, in particular, Growth differentiation factor 11 (GDF11), as an indicator of responsiveness of a patient to the treatment, efficacy of the treatment, or appropriate dosage for the treatment with an activin type II receptor inhibitor.

Abstract: The present invention is in the technical field of transplantation, and more particularly relates to the prognosis of a renal transplantation, and to the prevention of renal ischemia and reperfusion injury (IRI) prior to renal transplantation. The invention is more particularly based on the findings that the circulating level of ferritin in a patient is predictive of the outcome of renal transplantation, and that an iron administration to said patient prior to transplantation can prevent IRI.

Abstract: Provided herein are methods for the treatment of anemia, wherein the methods comprise administration of antagonists of Growth differentiation factor 11 (GDF1 1; also known as bone morphogenetic protein 11 (BMP11)) to a subject in need of the treatment.

Abstract: The present invention relates to methods, pharmaceutical compositions and kits for use in the treatment of Adult T-cell leukemia/lymphoma. More particularly, the present invention relates to a combination of an interferon, an arsenic compound and a reverse transcriptase inhibitor for the treatment of Adult T-cell leukemia/lymphoma.

Abstract: The present disclosure relates to antagonists of transferrin receptor and compositions and methods of use of said antagonists for treating pathological disorders such as thalassemia disorders

Abstract: Methods and compositions for the treatment of ?-thalassemia are provided. Methods and compositions restore or increase erythrocyte maturation in individuals afflicted with ?-TM by preventing proteolysis of GATA-1 protein. Screening methods for identifying agents which bind heat shock protein 70 (HSP-70) and inhibit HSP-70 ?-globin binding, but which allow GATA-1 protein-HSP-1 binding in a manner that prevents GATA-1 proteolysis.

Abstract: The present invention relates to methods, pharmaceutical compositions and kits for use in the treatment of Adult T-cell leukemia/lymphoma. More particularly, the present invention relates to a combination of an interferon, an arsenic compound and a reverse transcriptase inhibitor for the treatment of Adult T-cell leukemia/lymphoma.

Abstract: The present invention relates to methods, pharmaceutical compositions and kits for use in the treatment of Adult T-cell leukemia/lymphoma. More particularly, the present invention relates to a combination of an interferon, an arsenic compound and a reverse transcriptase inhibitor for the treatment of Adult T-cell leukemia/lymphoma.