National Center for Advancing and Translational Sciences Genetic and Rare Diseases Information Center, a program of the National Center for Advancing and Translational Sciences

Familial pulmonary arterial hypertension leucopenia and atrial septal defect


Other Names:
Familial pulmonary arterial hypertension, leucopenia and ASD; Familial PAH, leucopenia and ASD

FDA-Approved Treatments

The medication(s) listed below have been approved by the Food and Drug Administration (FDA) as orphan products for treatment of this condition. Learn more orphan products.

  • Selexipag (Brand name: Uptravi) - Manufactured by Actelion Ltd
    FDA-approved indication: For use of Uptravi (Selexipag) Tablets, 200, 400, 600, 800, 1000, 1200, 1400, and 1600 mcg for treatment of pulmonary arterial hypertension (PAH, WHO Group I) to reduce the risks of disease progression and hospitalization for PAH.
    National Library of Medicine Drug Information Portal

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