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Diseases

Genetic and Rare Diseases Information Center (GARD)

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Paroxysmal nocturnal hemoglobinuria


Other Names for this Disease
  • Marchiafava-Micheli disease
  • PNH
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Treatment


Clinical Trials & Research for this Disease

  • ClinicalTrials.gov lists trials that are studying or have studied Paroxysmal nocturnal hemoglobinuria. Click on the link to go to ClinicalTrials.gov to read descriptions of these studies.
  • The Rare Thrombotic Diseases Consortium aims to improve the lives of people with antiphospholipid antibody syndromes, heparin-induced thrombocytopenia, paroxysmal nocturnal hemoglobinuria, catastrophic antiphospholipid antibody syndrome (thrombotic storm), and thrombotic thrombocytopenic purpura (TTP) through research.

    For more information, please contact:

    Sharon Hall
    Research Analyst
    Hemostasis and Thrombosis Research Center
    Duke University Health System
    P.O. Box 3422 DUMC
    315 Trent Dr. Rm 273
    Durham, NC 27710
    Lab: 919-668-6329
    Office: 919-681-9565
    Fax: 919-681-6531
    E-mail: Sharon.hall@duke.edu

Medical Products

The medication(s) listed in the table(s) below have been approved by the Food and Drug Administration (FDA) for treatment of this condition. The FDA Office of Orphan Products Development designates "orphan products" for those that treat rare diseases affecting fewer than 200,000 Americans. The table(s) below may not be an exhaustive list of drugs or products used to treat this condition. There may be other products available that are not considered orphan products. To search for all FDA approved drugs, visit Drugs@FDA. You can find orphan products used to treat other conditions by searching the Orphan Drug Product Designation database.


Generic Name Eculizumab
Trade Name
(Manufacturer Name)
Soliris®
(Alexion Pharmaceuticals, Inc.)
Indication
The FDA has approved this product to be used in this manner.
Treatment of paroxysmal nocturnal hemoglobinuria to reduce hemolysis
More Information about this product Drug Information Portal