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  1. What are the treatments for PNH?

    ... This reduces hemolysis , the destruction of red blood cells. Bone marrow/stem cell transplantation (BMT/SCT):  A procedure that replace your unhealthy ...

    FAQ last updated 03/15/2016 - 11:32pm.

  2. Jeffrey Pu, MD, PhD

    ... clonal disorder originating from a multipotent hematopoietic stem cell (HSC) acquiring a PIG-A gene mutation . PIG-A ... 60% of AA patients also harbor small populations of PNH-like cells (0.01-10%). It was observed that: 1) 10-20% of AA patients harboring ...

    Grant Recipient last updated 10/01/2013 - 1:51pm.

  3. T-cell Depleted Alternative Donor Transplantation

    ... disease (GVHD) in patients receiving a stem cell transplant from an alternative (unrelated and mismatched related) ... and post-transplant infections. Patients requiring stem cell transplants for either malignant (cancerous) or non-malignant disease ... Device: CliniMACS® (T cell depletion) Stem cells will be collected from donors after they receive Granulocyte ...

    Clinical Trial last updated 04/29/2016 - 3:38pm.

  4. Crenolanib in Combination With Sorafenib in Patients With Refractory or Relapsed Hematologic Malignancies

    ... salvage therapy, or has relapsed after hematopoietic stem cell transplant (HSCT). Participant disease tested positive for ... on Day 8 of the first cycle. If they do not have leukemia cells in their spinal fluid, they will receive only one IT chemotherapy per ...

    Clinical Trial last updated 06/06/2016 - 10:04am.

  5. Allogeneic Bone Marrow Transplantation Using Less Intensive Therapy

    ... RATIONALE: A peripheral stem cell transplant may be able to replace blood-forming cells that were destroyed by chemotherapy and radiation therapy, or ...

    Clinical Trial last updated 05/02/2016 - 2:20pm.

  6. TCRαβ+/CD19+ Depleted Haploidentical HSCT + Zoledronate

    ... transplantation with a haploidentical donor peripheral blood stem cell graft depleted of TCRαβ+ cells and CD19+ cells in conjunction with the immunomodulating drug, ...

    Clinical Trial last updated 04/29/2016 - 4:35pm.

  7. Policymakers Act on Rare Disease Community’s Behalf

    ... 16, Congress finalized the reauthorization of a bill on stem cells sponsored by U.S. Rep. Chris Smith (R-NJ), who also introduced the ...

    Page last updated 01/08/2016 - 2:39pm.

  8. Sirolimus and Mycophenolate Mofetil in Preventing GVHD in Patients With Hematologic Malignancies Undergoing HSCT

    ... with hematologic malignancies undergoing hematopoietic stem cell transplant (HSCT). Biological therapies, such as sirolimus and ... suppress the immune system in different ways and stop tumor cells from growing. Giving sirolimus and mycophenolate mofetil after ...

    Clinical Trial last updated 05/03/2016 - 11:35am.

  9. Multicenter Phase II of CD26 Using Sitagliptin for Engraftment After UBC Transplant

    ... or partially matched UCB unit with >/= 2.5 x10^7 nucleated cells/kg of recipient weight at the time of cryopreservation. No current ... . Prior allogeneic or autologous hematopoietic stem cell transplant in the last 6 months. Patients who are taking ...

    Clinical Trial last updated 06/06/2016 - 3:37pm.

  10. Multicenter Phase II of CD26 Using Sitagliptin for Engraftment After UBC Transplant

    ... or partially matched UCB unit with >/= 2.5 x10^7 nucleated cells/kg of recipient weight at the time of cryopreservation. No current ... . Prior allogeneic or autologous hematopoietic stem cell transplant in the last 6 months. Patients who are taking ...

    Clinical Trial last updated 06/03/2016 - 2:54pm.