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Saturday, August 30, 2025

Asklepion Pharmaceuticals


Asklepion Pharmaceuticals products


CHOLBAM (cholic acid, oral)
Indication: Treatment of bile acid synthesis disorders—due to single enzyme defects and as adjunctive therapy in peroxisomal disorders (e.g., Zellweger spectrum)
Status: FDA-approved as an oral therapy

Turnobi (intravenous L-citrulline)
Indications and Clinical Trials:

  • Prevention of acute lung injury in pediatric patients undergoing cardiopulmonary bypass for congenital heart defect surgery (Phase III clinical trial underway under FDA Special Protocol Assessment)

  • Treatment of vaso-occlusive crisis in pediatric and young adult patients with sickle cell disease (Phase I/II trial conducted)




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