Asklepion Pharmaceuticals is dedicated to the development of pharmaceutical products specifically aimed at treating pediatric patients with rare diseases. The company focuses on discovering and commercializing therapies for conditions that currently have limited or no treatment options. One of their key investigational candidates is intravenous citrulline, which is being studied for its potential to prevent complications associated with acute lung injury in pediatric patients undergoing cardiopulmonary bypass surgery for congenital heart defects. This research is particularly significant given the challenges posed by postoperative pulmonary dysfunction, which can arise from decreased levels of nitric oxide due to reduced availability of citrulline during surgery.

The company has received Orphan Drug Designation from the U.S. FDA for its investigational therapies, highlighting its commitment to addressing the needs of patients with rare conditions. Asklepion's clinical trials, including a Phase III trial for L-citrulline, aim to provide evidence for the efficacy and safety of their treatments. The focus on pediatric clinical research underscores their mission to improve outcomes for children facing complex health challenges, particularly those related to congenital heart defects and the associated surgical interventions. Through these efforts, Asklepion Pharmaceuticals seeks to enhance the therapeutic landscape for rare pediatric diseases.

United States

We are a pediatric rare disease pharmaceutical development company.

Asklepion Pharmaceuticals, LLC focuses on the discovery, development, and commercialization of pharmaceutical products for pediatric patients with few therapeutic options, due to the rarity of their conditions. Our pipeline currently focuses on rare and critical diseases in children.


Asklepion Pharmaceuticals

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