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  1. San Diego Family Takes Action for Awareness

    ... have – and he was put on azacitidine. We also learned a clinical study of oral azacitidine – taken by mouth, in pill form. We were ... biopsy to see if prospective patients are qualified for the trial. However, because he remained in high-risk status he wasn’t qualified, ...

    Patient Chronicle last updated 10/26/2016 - 11:42am.

  2. Design and rationale of the QUAZAR Lower-Risk MDS (AZA-MDS-003) trial: a randomized phase 3 study of CC-486 (oral azacitidine) plus best supportive care vs placebo plus best supportive care in patients with IPSS lower-risk myelodysplastic syndromes and po

    ... modifier azacitidine . In an expanded phase 1 trial, CC-486 demonstrated clinical and biological activity in patients with International Prognostic ...

    Research Article last updated 06/23/2016 - 10:17am.

  3. Interviews with the Experts: Dr. Bart Scott on PNH

    ... does. Because it is so rare, it’s hard to design a clinical trial to study the disease in a comprehensive manner. So few have it, and ...

    Interview last updated 09/23/2016 - 2:33pm.

  4. Self-injected PNH therapy on the horizon

    ... positive interim data from the first cohort in its phase Ib trial that show maintenance of complement inhibition using Coversin in healthy ... diseases, and Alexion presented the first clinical data for its own next-generation complement inhibitors. Overall, we ...

    Article last updated 07/07/2016 - 9:40am.

  5. AK579 (COBALT)

    For more details on this clinical trial, including contact information, please see this trial’s listing on ... of meningitis prophylaxis Participation in other clinical trials within 4 weeks of signing the consent form Known allergy to ...

    Clinical Trial last updated 09/14/2016 - 10:39am.

  6. Major news in BMT with half-match donors

    ... and the primary investigator of many UT Southwestern clinical trials related to bone marrow transplantation. Mr. Dandridge enrolled in one such UT Southwestern clinical trial , studying a therapy called AG-221, developed by Agios Pharmaceuticals ...

    Article last updated 06/14/2016 - 3:02pm.

  7. NHLBI profiles a member of the Foundation’s patient community.

    ... of Health (NIH), asking if she could be a part of the trial. She learned that to be eligible, she first needed to be a ... as much about their disease as possible — as well as about clinical trials being conducted at the National Institutes of Health.” ...

    Article last updated 06/13/2016 - 1:51pm.

  8. A Biomarker-Directed Phase 2 Trial of SY-1425 in Patients With Acute Myeloid Leukemia or Myelodysplastic Syndrome

    For more details on this clinical trial, including contact information, please see this trial’s listing on clinicaltrials.gov: ...

    Clinical Trial last updated 08/24/2016 - 11:20am.

  9. Coversin in Paroxysmal Nocturnal Haemoglobinuria (PNH) in Patients With Resistance to Eculizumab Due to Complement C5 Polymorphisms

    For more details on this clinical trial, including contact information, please see this trial’s listing on ... the efficacy, appropriateness or suitability of any clinical trial listed on this website. Pharmaceutical company sponsored ...

    Clinical Trial last updated 08/26/2016 - 11:59am.

  10. Interviews with the Experts: Side Effects of PNH Treatment

    ... reported side effects of eculizumab in the PNH randomized trial were: headache, nasopharyngitis, back pain, and nausea. Interestingly, ... of Chicago.  She is board certified in anatomic and clinical pathology, internal medicine, and hematology, and board eligible in ...

    Interview last updated 08/31/2016 - 8:37am.