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  1. New treatment approved for certain high-risk AML

    ... The U.S. Food and Drug Administration today approved Vyxeos for the treatment of adults with two ... Breakthrough Therapy designations. Vyxeos also received Orphan Drug designation, which provides incentives to assist and encourage ...

    Article last updated 08/30/2017 - 7:18am.

  2. Resources to Help

    ... PHARMACEUTICAL COMPANY PATIENT ASSISTANCE (drug specific) . INSURANCE AND CO-PAY ASSISTANCE . ... and other languages about more than 950 rare diseases and orphan drugs. World Marrow Donor Association The World ...

    Topic section last updated 11/15/2017 - 2:31pm.

  3. Rare disease topic tackled by World Economic Forum

    ... systems. A rare disease, sometimes referred to as an orphan disease, is any disease that affects a small percentage of the general ... systems. A rare disease, sometimes referred to as an orphan disease, is any disease that affects a small percentage of the general ... and regulatory authorities, such as the Food and Drug Administration (FDA) and European Medicines Agency (EMA), to design a ...

    Article last updated 02/06/2017 - 2:59pm.

  4. Resources to Help

    ... PHARMACEUTICAL COMPANY PATIENT ASSISTANCE (drug specific) . INSURANCE AND CO-PAY ASSISTANCE . ... and other languages about more than 950 rare diseases and orphan drugs. World Marrow Donor Association The World ...

    Topic section last updated 09/28/2017 - 11:58am.

  5. Patrizia Ricci, PhD

    ... based in Europe) we have applied for the status of Orphan Drug to competent authorities. Indeed, without further discussion AMY-101 ...

    Grant Recipient last updated 09/14/2016 - 2:42pm.

  6. Medical Resources

    ... and Harvard Medical Schools.  Drug Information Food & Drug Administration (FDA) (888) ... medication information. Office of Orphan Products Development Dedicated to promoting the development of ...

    Page last updated 08/20/2015 - 8:34am.

  7. Steven C. Groft, Pharm.D.

    ... Dr. Groft has been focusing attention on rare and orphan diseases like bone marrow failure . His leadership and vision ... Highlights of his work include time at the Food and Drug Administration in the Office of Orphan Products Development and at the ...

    Leadership Award last updated 07/11/2016 - 3:20pm.

  8. Chao-Yie Yang, MD, PhD

    ... (AMY-101) based on the discovery of compstatin has received orphan status in Europe and US for treatment of PNH in 2014. AMY-101 inhibits ... in monkey via intravenous injection. Currently, no oral drug is available to treat PNH. In this proposed work, we will discover and ...

    Grant Recipient last updated 08/09/2016 - 1:02pm.