Pipeline
Our goal: To develop small molecules to improve the lives of patients with genetically defined rare diseases.

DBA: Diamond Blackfan Anemia; BMFS: Bone Marrow Failure Syndromes
Our goal: To develop small molecules to improve the lives of patients with genetically defined rare diseases.

DBA: Diamond Blackfan Anemia; BMFS: Bone Marrow Failure Syndromes